ADIS DRUG EVALUATION Drugs 1998 Sep; 56 (3): 429-445 0012-6667/98/0009-0429/$17,00/0 Adis International Limited. All rights reserved. Domperidone A Review of its Use in Diabetic Gastropathy Amitabh Prakash and Antona J. Wagstaff Adis International Limited, Auckland, New Zealand Various sections of the manuscript reviewed by: A.P. Braun, Diabetes Center of New Jersey, Muhlenberg Regional Medical Center, University of Medicine and Dentistry of New Jersey, Rutgers, New Jersey, USA; U. Bonucceli and P. Del Dotto, Dipartimento di Neuroscienze Dell'Universita' di Pisa, Pisa, Italy; M. Hongo, Department of Comprehensive Medicine, Tokohu University School of Medicine, Sendai, Japan; M. Horowitz, Department of Medicine, Royal Adelaide Hospital, Adelaide, South Australia, Australia; K.L. Koch, Department of Medicine, Section of Gastroenterology and Hepatology, The Milton S. Hershey Medical Center, Hershey, Pennsylvania, USA; R.A. Kozarek, Department of Medicine, Section of Gastroenterology, Virginia Mason Medical Center, University of Washington, Seattle, Washington, USA; P-H. Nilsson, Department of Internal Medicine, Central Hospital, Växjö, Sweden; D.K, Sommers, Department of Pharmacology, University of Pretoria, Pretoria, South Africa; M. Velasco, Clinical Pharmacology Unit, Vargas Medical School and Vargas Hospital, Caracas, Venezuela; J.W. Wiley, Department of Internal Med'cine, Division of Gastroenterology and Gastroenterology Research Unit, The University of Michigan Medical Center, Ann Arbor, Michigan, USA. Data Selection Sources: Medical literature published since 1966 on domperidone, identified using AdisBase (property database of Adis International, Auckland, New Zealand), Medline and EMBASE. Additional references were identified from the reference lists of published articles. Bibliographical information, including contributory unpublished data, was also requested from the company developing the drug. Search strategy: AdisBase search term were 'domperidone', 'gastrokinetics', 'diabetes mellitus', 'diabetic complications' and 'gatroparesis' Medline and EMBASE search terms were 'domperidone' ‘diabetes’, ‘gastroparesis or stomach-disease’ and ‘domperidone –pharmacodynamic and – pharmacokinetic’. Searches were updated 17 Jul 1998. Selection: Studies in patiets with diabetes mellitus with gastropathy who received domperidone. Inclusion of studies was based mainly on the methods section of the trials. When available, large, well controlled trials with appropriate statistical methodology were preferred. Relevant pharmacodynamic and pharrnacokinetic data were also included. Index terms: Domperidone, diabetic complications, gastroparesis, pharmacokinetics, pharmacodynamics, therapeutic use, tolerability, drug interactions, dosage and administration and quality of life. Contents Abstract ..................................................................................................................................... 430 1. Diabetic Gastropathy .................................................................................................................... 431 2. Pharmacodynarnic Properties ....................................................................................................... 432 2.1 Mechanism of Action ................................... . .......................................................................432 2.2 Gastrointestinal Effects ............................................................................................................432 2.2.1 Effects on the Stomach and Duodenum .......................................................................432 2.3 Endocrine Effects .....................................................................................................................434 2.3.1 Plasma Prolactin Levels ....................... ..........................................................................434 2.3.2 Plasma Insulin Levels ........................................... . ......................................................... 434 2.3.3 Effects on Other Hormones ............................................................................................ 434 2.4 Other Effects ............................................................................................................................ 435 3. Pharmacokinetic Properties ........................................................................................................... 435 3.1 Absorption and Distribution ...................................................................................................... 435 430 4. 5. 6. 7. Prakash & Wagstaff 3.2 Metabolism and Excretion ................................................................................................. .... 435 3.3 Drug Interactions ...................................................................................................................... 436 Clinical Efficacy .............................................................................................................................. 436 4.1 Symptomatic Improvement ................................. .................................................................. 436 4.1.1 Short Term Trials .............................................................................................................. 436 4.1.2 Long Term Trials .............................................................................................................. 439 4.1.3 Trials in Patients with Symptomatic Gastropathy Refractory to Other Agents ............... 439 4.2 Effects on Quality of Life .......................................................................................................... 439 4.3 Other End-points .............................................................................. ...................................... ..440 Tolerability ....................................................................................................................................... 440 Dosage and Administration .......................................................................................................... ...441 Place of Domperidone in the Management of Diabetic Gastropathy ......................................... 441 Abstract Domperidone is a selective antagonist at peripheral dopamine D2 receptors, with gastroprokinetic and antiemetic properties. It increases the frequency and duration of antral and duodenal contractions, thus decreasing/improving transit time of food through the gastrointestinal tract. Gastric emptying of liquids and solids is significantly improved with oral domperidone 40 to 120 mg/day in patients with diabetic gastropathy. Oral domperidone 40 to 80 mg/day significantly decreased the severity of symptoms of gastropathy from baselim values in 66 to 88% of patients with type 1 (insulin-dependent) or insulin-requiring diabetets mellitus, Double-blind withdrawal of domperidone from patients who had responded previously led to greater deterioration of symptoms in patients with delayed gastric emptying than in those who continued receiving the drug. Quality of life was significantly improved in patients who showed a symptomatic response to domperidone. The administration of domperidone 40 to 120 mg/day significantly reduced hospitalisation rates in patients with gastropathy. The symptomatic improvement with domperidone 80 mg/day was similar to that seen with cisapride 40 rng/day or metoclopramide 40 mg/day, and therapeutic benefits seen in symptoms of .gastropathy were maintained with domperidone for up to 12 year:-. Domperidone 40 to 80.mg/day may be effective in patients who are refractory to metoclopramide, and a combination of domperidone 80 mg/day with cisapride 80 mg/day may improve some symptoms in patients who do not respond to either agent alone. Domperidone 40 to 120 mg/day was well tolerated for periods up to 12 years in trials in patients with diabetic gastropathy. Adverse events with domperidone 80 mg/day were similar to those seen in placebo recipients and significantly fewer than in patients receiving metoclopramide 40 mg/day. Although significant elevation of plasma prolaclin levels (unrelated to dosage and. duration of treatment) occurred in all domperidone recipients, prolacUn-related adverse events were observed in only 10 to 20% of patients. Conclusions: The available data suggest that domperidone 40 to 80 mg/day is an effective agent for the management of symptoms of gastropathy in patients with type 1 diabetes mellitus. In addition, it may provide symptom improvement in patients with gastropathy refractory to other gastroprokinetic agents. Domperidone maintains efficacy in the long term (up to 12 years) and appears to have a better tolerability profile than metoclopramide 40 mg/day. © Adis International Limited. All rights reserved. Drugs 1998 Sep; 56 (3) Doperidone: A Review 1. Diabetic Gastropathy The term 'gastroparesis diabeticorum' was coined by Kassander 40 years ago to describe asymptomatic gastric retention in patients with diabetes mellitus; delayed gastric emptying of solids and liquids may occur in 50% of diabetic patients.[1] Gastric emptying was studied in 12 patients with type 1 diabetes mellitus and in 22 healthy volunteers.[2] All the evaluated parameters, including solid lag phase (57 vs 35 min, p < 0.001), solid retention at 100 min (79 vs 29%, p < 0.001) and liquid 50% emptying time (50 vs 18 min, p < 0.001), showed significant impairment of gastric emptying in diabetic patients compared with volunteers. However, the presence of delayed gastric emptying or gastroparesis has shown poor correlation with the prevalence of upper gastrointestinal symptoms in patients with diabetes mellitus. A metaanalysis of trials in patients with gastropathy who had been treated with oral prokinetic agents found no association between symptomatic improvement and accelerated gastric emptying.[3] In view of the fact that many diabetic patients with symptoms of 'gastroparesis' have normal gastric emptying[4] and that up to 50% of diabetic patients with marked delay in gastric emptying may have few or no symptoms,[5] terms such as 'diabetic dyspepsia'[6] or 'diabetic gastropathy' [7,8] have been favoured to describe the clinical syndrome. The precise prevalence of symptomatic gastropathy in patients with diabetes mellitus is unknown but 76% of 136 diabetic patients reported gastrointestinal symptoms on being specifically questioned.[9] The symptoms of gastropathy in patients with diabetes mellitus appear to be multifactorial in origin. The various pathophysiological changes such as decreased motor activity of the stomach (fundic or antral activity, phase three interdigestive motor activity, migrating motor complex or pylorospasm) have been reviewed elsewhere, as have the underlying pathogenetic mechanisms such as autonomic neuropathy, abnormalities in gut hormone levels, hyperglycaemia, electrolyte imbalances, changes in secretion of insulin and glucagon, and © Adis International Limited. All rights reserved. 431 micro- and macroangiopathy of the stomach.[10,11] Recent studies have highlighted the importance of blood glucose levels in the modulation of gastric electrical and motor activity, as well as in the causation of upper gastrointestinal symptoms in patients with type 1 diabetes mellitus.[12-17] Symptoms of gastropathy include nausea and vomiting, epigastric burning, early satiety, belching, regurgitation, postprandial fullness, brittle diabetes, malnutrition, weight loss and symptoms related to erratic drug absorption.[1] Diabetic gastropathy is a chronic condition and despite glycaemic control and dietary manipulation, it has been estimated that 80% of patients require long term maintenance therapy with prokinetic and/or antiemetic agents.[18] Domperidone (fig. 1) is a benzimidazole which is structurally related to the butyrophenones. Oral domperidone is effective in the management of emesis due to a wide variety of causes, including that induced by anticancer agents. [19] Recently, domperidone has also been found to be effective in the treatment of patients with idiopathic dyspepsia. [20-22] Although the prevalence of symptomatic gastropathy in patients with type 1 (insulin dependent) diabetes mellitus may be similar to that in patients with type 2 (non-insulin-dependent) diabetes mellitus,[5] almost all published trials involving domperidone have evaluated the efficacy of the drug in patients with type 1 diabetes mellitus with symptoms of gastropathy (section 4). The focus of this review, therefore, will be on the efficacy of domperidone in the management of patients with diabetes mellitus and gastropathy. Drugs 1998 Sep; 56 (3) 432 Prakash & Wagstaff 2. Pharmacodynamic Properties 2..1 Mechanism of Action The primary mechanism by which domperidone exerts its pharmacological actions is believed to be selective antagonism of peripheral dopaminergic D2 receptors.[23] Other suggested mechanisms include increased acetyicholine release[24] and inhibition of cholinesterase activity[25] Table I provides an overview of studies investigating these effects. 2.2 Gastrointestinal Effects A single oral close of domperidone 30mg significantly reduced the mouth to caecum transit time (from 89 to 63 min, p < 0,01) as measured by a breath H2 test in 6 healthy volunteers.[31] The effects of oral domperidone on lower oesophageal sphincter pressure (LOSP) are equivocal and may be dose-dependent. LOSP was increased, by about 15mm Hg over placebo values, after 40 to 90 minutes in 9 patients receiving a single high dose of domperidone l00mg[32] A significant (p < 0.01) increase in LOSP from baseline values was also reported in healthy volunteers (numbers not stated) and 47 patients with gastro-oesophageal reflux disease receiving domperidone (dose not stated).[33] However, in 2 other studies in 10 healthy volunteers receiving a single dose of domperidone 20mg[34] or 22 patients with reflux oesophagitis receiving domperidone 20 mg/day for 14 days in addition to antacids and alginate,[35] no change in LOSP from placebo values was seen. Furthermore, in contrast to its inhibitory effect on upper gastrointestinal motility, dopamine has a stimulatory effect on colonic motor activity.[36] Intravenous domperidone 0.4 mg/kg significantly inhibited increased rectosigimoid colon motor activity after intravenous dopamine 50 g/kg and the early and late increase in motor activity occurring postprandially in 12 healthy volunteers.[36] Oral domperidone l00mg, administered as 5 doses over 36 hours, did not alter the stress-induced inhibition of fasting migrating motor complexes in the small bowel in 7 healthy volunteers.[37] © Adis International Limited. All rights reserved. 2.2.1 Effects or the Stomach and Duodenum Effects or Pressure Waves Two early trials midied the effect of domperidone on gastric and duodenal molility by measuring intraluminal pressure waves.[38,39] Intravenous domperidone 8mg increased the duration of contractions (from 3.6 to 9.4 seconds) in the distal part of the stomach (antrum) in 10 healthy volunteers,[38] and 20mg increased the duration and frequency of antral contractions in 30 patients with dyspepsia.[39] Intravenous domperidone 8mg also increased the frequency (from 1.0 to 3.2 per minute), amplitude (from 9.0 to 16.0mm Hg) and duration (from 3.0 to 4.5 seconds) of duodenal contractions in 10 healthy volunteers.[38] However, there appeared to be no effect on the overall frequency of duodenal contractions in 30 patients with dyspepsia after intravenous domperidone 20mg, despite decreases in the number of isolated duodenal contractions in those patients with spontaneous duodenal activity (>5 contractions per 10 min).[39] Effects on Gastric Emptying A review of early studies concluded that intravenous domperidone 20mg appeared to increase gastric emptying in both healthy volunteers and patients with dyspepsia or delayed gastric emptying from other causes.[19] Single oral doses of domperidone 30mg, but not 1.5mg, increased gastric emptying of liquids in patients with delayed emptying and slowed emptying in those in whom it was Drugs 1998 Sep; 56 (3) Domperidone: A Review pathologically accelerated. [14] Similarly, single doses of domperidone 40mg significantly increased solid emptying rates in patients with diabetic gastropathy.[2] However, in patients with diabetic gastropathy who had been receiving domperidone 60 ing/day for up to 51 days, the mean gastric emptying rate of solids after a single dose of domperidone 40mg was similar to that with placebo.[2] Patients with diabetic gastropathy receiving oral domperidone 40 to 120 mg/day for 1 to 31 months. had improved solid gastric emptying rates (9 to 34%) [table II]. Improvements in gastric emptying reached statistical significance in only 2 studies, but this may be a result of small patient numbers in most trials. After 4 weeks, domperidone 80 mg/day and cisapride 40 mg/day decreased gastric emptying time to a similar extent (from 146 to 83 min vs from 105 to 79 min) in patients with diabetic (n = 9) or idiopathic (n = 1) gastroparesis in an open-label trial.[45] Electrogastrographic Effects Surface recording of myoelectrical activity of the stomach can be used to evaluate patients with symptoms of gastropathy.[11] Electrogastrographic (EGG) studies in patients with diabetes mellitus with symptoms of gastropathy showed abnormalities of gastric electrical rhythms irrespective of the presence or absence of delayed gastric empThe EGG recordings from 23 patients © Adis International Limited. All rights reserved. 433 with type 1 diabetes mellitus and symptoms of gastropathy (14 with and 9 without delayed gastric emptying) showed a higher proportion of waves with a frequency of 1 to 2 cycles per minute (cpm) and a lower proportion with a frequency of 3 cpm than seen in 12 healthy controls.[48] The results of 2 small trials suggest that domperidone is effective in converting the disturbed gastric myoelectrical rhythm towards normal EGG wave patterns in patients with type 1 diabetes mellitus.[41,49] After a 4-week single-blind administration of domperidone (dosage not stated) to 23 patients with type 1 diabetes mellitus and gastropathy, 16 symptomatic responders (9 with delayed and 7 with normal gastric emptying) were randomised to receive either domperidone or placebo (domperidone withdrawal) for a further 4 weeks.[49] Among patients with delayed gastric emptying, EGG recordings from those who continued to receive domperidone (n = 5) showed significantly increased 3 cpm waves (p < 0.05) and significantly decreased 1 to 2 cpm waves (p < 0.01) compared with those who received placebo (n = 4).[49] EGG recordings from patients with normal gastric emptying receiving domperidone (n = 4) showed a significant decrease in 4 to 9 cpm waves (p < 0.01) and a nonsignificant increase in 3 cpm waves compared with those receiving placebo (n = 3).[49] After 6 months, 4 of 6 patients with type 1 diabetes Drugs 1998 Sep; 66 (3) Prakash & Wagstaff 434 mellitus and symptoms of gastropathy (refractory to other prokinetic and antiemetic agents) receiving domperidone 80 mg/day showed normal 3 cpm EGG wave patterns in an open-label trial.[41] 2.3 Endocrine Effects 2.3.1 Plasma Prolactin Levels Administered orally or intravenously, domperidone is a potent stimulant of prolactin release, although peak prolactin levels are consistently higher in females than in males.[19] The administration of a single oral or intravenous dose of domperidone 10 or 20mg significantly increased plasma and serum prolactin levels from pretreatment values (from 2.5 to 9,5 g/L to 19 to 125 g/L) in small numbers (n = 5 to 17 per group) of healthy volunteers, or patients with type 1 diabetes mellitus or acute or chronic schizophrenia.[150-55] Serum prolactin levels peaked 30 minutes after intravenous administration and 60 minutes after intramuscular or oral administration of domperidone; prolactin levels returned to baseline values after 8 hours.[51] Oral domperidone 60 mg/day for 4 weeks also resulted in 5-fold increases in prolactin levels over baseline values in 4 patients with gastro-oesophageal reflux disease[51] and in 49 patients (diagnoses not provided).[56] There appears to be no relationship between the dosage of domperidone or the duration of treatment and the increase in plasma prolactin levels.[51,56] However, the duration of type 1 diabetes mellitus may influence the prolactin response to domperidone. The increase in prolactin levels after intravenous domperidone l0mg was significantly lower in 8 patients with type 1 diabetes mellitus of 11 to 18 years' duration (3.5 times baseline values) than in 7 healthy controls or 8 patients with type 1 diabetes mellitus of 1 to 9 years' duration (both about 5 times baseline values).[52] The investigators suggest that reserves of prolactin which are available for acute release are reduced in patients with long-standing type 1 diabetes mellitus.[52] Age of the recipient may also affect the prolactin response to a single oral dose of domperidone. The prolactin response in elderly healthy volunteers receiving domperidone 20mg was sig© Adis international Limited. Aii rights reserved. nificantly lower than in young healthy volunteers, but the response was similar in young patients with acute schizophrenia and elderly patients with chronic schizophrenia receiving domperidone 20mg.[54] 2.3.2 Plasma Insulin Levels Although some investigators have suggested a role of dopamine and its receptor subtypes in regulating insulin secretion,[57] available data are inconclusive. Intravenous infusion of dopamine 0.5 to 3 g/kg min for 30 minutes did not alter the blood glucose or plasma insulin levels in 7 healthy volunteers.[58] However, a similar infusion of dopamine significantly increased the plasma insulin levels from baseline values (86.74 vs 59.48 pmol/L, p < 0.01) in 19 hypertensive patients who had been pretreated with labetalol 0.8 to 1.2 g/day for 7 days.[57] A dopamine-induced rise in plasma insulin levels was not observed in these patients after pretreatment with domperidone 40 mg/day for 7 days.[57] The effects of domperidone on glucose-induced insulin secretion are equivocal. While increases in plasma insulin levels after an oral glucose load of 75g were similar after a single oral dose of domperidone 10mg or placebo in 9 volunteers (330 to 373 pmol/L, approximate values from graph),[55] plasma insulin levels after intravenous glucose 0.5 mg/kg were higher in 8 volunteers who received oral domperidone l00mg 90 minutes beforehand than in 8 volunteers who received placebo (244 vs 172 pmol/L after 10 minutes, values from graph).[59] 2.3.3 Effects on Other Hormones The effects of domperidone on plasma aldosterone levels have been investigated in several trials involving small groups (6 to 10 per trial) of healthy volunteers[50,60,61] or in 5 patients with essential hypertension.[50] While intravenous domperidone l0mg appeared to have little effect on plasma aldosterone levels in one trial,[50] an intravenous dose of 40mg and an oral dose of 20mg in two further trials decreased the plasma levels of aldosterone from baseline values by about 15%.[60,6l] Administration of oral domperidone 20mg or intravenous domperidone 10 to 20mg did not apDrugs 1998 Sep; 56 (3) Domperidone: A Review 435 Table III. Overview of the pharmacokinetic properties of oral domperidone in healthy volunteers Domperidone 10mgsd 60mga sd 60mgb sd No. of volunteers 5 7 4 F(%) Mean Cmax (g/L) 17.6 12.7 23 80 23.6 65 12-18 tmax (h) 0.5 0.5 2 AUC (g/L • h) t½ (h) 58 249 463 0.5-1.0 10mgqid 10mg qidc 20mg qidc 24 11.35 1.14 24 13.86 1.09 157.36 163.81 40mg qidc 24 14.13 1.23 181.26* Reference 67 67 67 7-16 68 69 69 69 a Tablet administered to fasting volunteers. b Tablet administered 90 minutes after a standard meal. c After 5 days and normalisation of individual values to the 40mg dose. AUC = area under the plasma concentration-time curve; Cmax = peak plasma concentration; F = systemic bioavailability; qid = 4 times daily; sd = single dose; t½ = elimination half-life; tmax = time to Cmax; * indicates significantly higher value than with 10 and 20 mg qid (p < 0.05). pear to affect plasma rcnin activity,[50,60,61] mealstimulated serum gastrin levels, [62] plasma levels of basal[63] or hypoglycaemia-stimulated[64] argininevasopressin, or plasma levels of adrenocorticotrophin or cortisol[50] in groups of 6 to 16 healthy volunteers or 5 patients with essential hypertension.[50] Oral domperidone 60 mg/day for 4 weeks significantly decreased plasma testosterone levels (855 vs 301 g/L, p < 0.05) but did not affect the levels of follicle-stimulating hormone or luteinising hormone in 49 male patients (diagnoses not stated).[56] The plasma levels of testosterone were similar to pretreatmerit values 2 weeks after domperidone withdrawal.[56] 2.4 Other Effects Domperidone, lOmg intravenously[50] or orally as a single close of 20mg'[61] or as 40 mg/day for 7 days[65] did not affect blood pressure or heart rate in healthy volunteers (n = 7 to 10 per trial)[50,61,65] or in 5 patients with essential hypertension.[50] A single oral dose of domperidone 60mg led to a small but significant increase in mean arterial pressure (92 vs 96mm Hg) in 6 healthy volunteers.[66] A single oral dose of domperidone 20 [61] or 60mg[66] or a total intravenous dose of 40mg'60' did not affect renal function in healthy volunteers.[60,61,66] 3. Pharmacokinetic Properties Since the publication of the previous review of domperidone in Drugs,[19] very few additional data have been published regarding the pharmacoki© Adis International Limited. All rights reserved. netic properties of domperidone in healthy volunteers (table III). The pharmacokinetic properties of the oral formulation of domperidone are overviewed in this section. There are no available data on the pharmacokinetic properties of domperidone in patients with type 1 diabetes mellitus with delayed gastric emptying, in whom drug absorption may be disturbed. 3.1 Absorption and Distribution In healthy volunteers, peak plasma domperidone concentrations (Cmax) were reached within 2 hours (tmax) after a single oral dose (table III). The systemic bioavailability of domperidone administered on an empty stomach was 12 to 18% and was increased to 23.6% in 1 study by administering the drug 90 minutes after a meal. Domperidone appears to undergo rapid 'first-pass' metabolism either in the gut wall or in the liver, as only 7% of the administered drug is excreted unchanged in the faeces.[70] About 92 to 93% of domperidone has been estimated to be bound to plasma proteins.[67] Animal data indicate that orally administered domperidone is widely distributed in the body.[7l] However, studies in healthy volunteers indicate that domperidone is unable to cross the blood-brain barrier.[50,62,63] 3.2 Metabolism and Excretion Oxidative N-dealkylation and hydroxylation to 2 major metabolites, neither of which has significant activity, seem to be the most important Drugs 1998 Sep; 56 (3) 436 Prakash & Wagstaff pathways for the metabolism of domperidone in humans.[70] Although the steady-stale pharmacokinetics (Cmax and tmax) were proportional to dos-age after domperidone 10, 20 and 40mg 4 times daily for 5 days, the highest dosage led to a signif-icantly higher area under the plasma concentration-time curve than the 2 lower dosages in a random-ised, double-blind, crossover trial in 24 healthy volunteers[69] (table III). The plasma elimination half-life (t½) of domperidone ranged from 7 to 16 hours after oral administration of 10mg 4 times daily.[68] The t½ is prolonged to up to 20.8 hours in patients with severe renal dysfunction (serum creatinine >530 mol/L).[19] However, since renal clearance is low compared with total plasma clearance (42 L/h), domperidone is unlikely to accumulate in patients with renal dysfunction.[19] After oral administration of 14C-domperidone 40mg to healthy volunteers, 31% of the radioactivity was excreted in urine (mostly within 24 hours) with only 0.43% of the administered dose present as unchanged drug; 66% of the radioactivity was excreted in the faeces over 4 days (6% of the administered dose as unchanged drug).[70] 3.3 Drug Interactions The tmax of pramipexole 0.25mg was significantly reduced with concomitant administration of domperidone 30mg in 12 healthy volunteers.[72] However, the pharmacokinetic disposition of aspirin 350mg was not altered by the addition of domperidone l0mg in a randomised, crossover trial in 6 healthy volunteers.[73] Similarly, pretreatment with domperidone 20mg did not alter any of the pharmacokinetic parameters of ropinirole 0.8mg in 9 healthy volunteers.[74] 4. Clinical Efficacy The efficacy of oral domperidone has been investigated in double-blind, single-blind and nonblind trials of 1 to 48 months' duration in patients with type 1 diabetes mellitus and symptoms of gastropathy. Domperidone 20mg 4 times daily appears to be effective in these patients and in patients © Adis international Limited. AH rights reserved. whose symptoms of gastropathy are unresponsive to other prokinetic or antiemetic agents. Much of the data discussed in this section are available only in abstract reports (see table IV). Also, many of the trials in patients with symptoms of gastropathy who had been treated with oral prokinetic agents have methodological limitations such as inadequate numbers of patients, lack of randomisation, lack of placebo control or poor definition of inclusion and exclusion criteria for patient selection.[3] Most of the trials were conducted in small numbers of patients with type 1 diabetes mellitus in whom blood glucose levels were not reported. The most common method of evaluating the efficacy of domperidone treatment was assessment of symptomatic improvement. This was achieved by measuring the reduction in total symptom score, reduction in intensity and frequency scores of symptoms, change in symptom score on a visual analogue scale, or physician- and/or patient-rated global evaluation. Although all trials evaluated severity of symptoms on an ordinal scale from 0 (absent) to 3 (severe), there were minor differences in the symptoms selected for evaluation (see table IV). Other efficacy parameters included hospitalisation rale, and improvement in quality of life, measured on the physical and mental component summary scores of the Medical Outcomes 36-item Short Form Health Survey (SF-36), compared with pretreatment values. 4.1 Symptomatic Improvement Most trials with symptomatic improvement as an end-point (table IV) included patients with type 1 diabetes mellitus who presented with symptoms of anorexia, early satiety, nausea, vomiting, abdominal distension/bloatedness and abdominal pain. 4.1.1 Short Term Trials As it is difficult to demonstrate the efficacy of prokinetic agents in patients with symptoms of gastropathy, the US Food and Drug Administration recommended an 'enriched' design for domperidone trials to identify treatment responders.[79] Drugs 1998 Sep; 56 (3) Domperidone: A Review Adis International Limited. All rights reserved. 437 Drugs! 098 Sep; 56 (3) 438 Following an initial nonblind or single-blind phase where all patients received domperidone, 4 trials compared the effects of domperidone with placebo (domperidone withdrawal) in a subsequent randomised single-blind or double-blind phase (table IV). In these trials, 66 to 88% of patients responded (good or excellent improvement in global assessment of symptoms or significant reduction in total symptom score) to domperidone 40 to 80 mg/day in the noncomparative phase. Those responding were randomly assigned to either continue domperidone 40 to 80 mg/day or receive placebo for a further period of 1 month in a randomised, single-blind or double-blind fashion (extended phase). Patients in whom domperidone was withdrawn in the extended trial phase (placebo-control) had a significant increase in median total symptom scores and a deterioration in global assessment rating compared with patients who continued to receive domperidone 40 to 80 mg/day (table JV). Prakash & Wagstaff Patients with delayed gastric emptying in whom domperidone was withdrawn in the extended phase deteriorated to a greater extent than those continuing with domperidone (p < 0.05) in 2 studies.[4,77] However, this effect was not observed in patients with normal gastric emptying (fig. 2). In a double-blind trial (n = 93), domperidone 80 mg/day and metoclopramide 40 mg/day improved total symptom scores to a similar extent and were considered equivalent in global evaluations.[78] Similarly, in a small open-label crossover trial, [45] domperidone 80 mg/day appeared to be equivalent to cisapride 40 mg/day in reducing total symptom score in 10 patients with diabetic gastropathy (ta-ble IV). Preservation of autonomic function in patient5 with type 1 diabetes mellitus with gastropathy may indicate a greater likelihood of sump improvement with domperidone. One small trial (n = 16) found that patients with type 1 diabetes mellitus with gastropathy who improved with dom- Fig. 2. Effect of domperidone (DOM) on symptoms of gastropathy in patients with insulin-requiring diabetes mellitus. Mean total symptom scores (TSS) were significantly decreased after DOM 80 mg/day for 4 weeks, in 208 of 269 patients with insulin-requiring diabetes mellitus with delayed or normal gastric emptying, in a single-blind enrichment phase. Treatment responders were randomised to either continue DOM or receive placebo for a further 4 weeks in a double-blind fashion.[4,75] * p < 0.05 vs placebo; ** p < 0.001 vs baseline. © Adis International Limited. All rights reserved. Drugs 1998 Sep; 56 (3) Domperidone: A Review peridone had better autonomic function than patients who did not respond to domperidone.[80] The investigators suggested that prior autonomic function testing in patients with diabetic gastropathy may be useful in defining patients whose symptoms are more likely to respond to domperidone. 4.1.2 Long Term Trials A sustained improvement in symptoms of gastropathy has been shown in groups of patients (n = 5 to 66 per trial) receiving domperidone for up to 12 years in several small, nonblind trials. [42-44,76,81,82] In 14 of 24 patients with diabetes mellitus, domperidone 80 mg/day significantly reduced the total symptom score at 12 months by 58.5% from baseline values (from 10.6 to 4.4, p < 0.0001).[8] In 2 trials which included 10[43] and 17144] patients with symptoms of gastropathy associated with diabetes, surgical procedure, scleroderma or unknown (idiopathic) aetiology,'43'44^ domperidone 40 to 120 mg/day over 21 and 48 months led to sustained, significant decreases in total symptom scores from baseline values, i.e. by 75% (from 8.4 to 2.1,. p < 0.05), and by 68% (from 4.1 to 1.3, p< 0.05), respectively. Domperidone 80 mg/day administered for an average of 1.73 years (range 0.3 to 8 years) improved symptoms of gastropathy in 63 of 66 patients (95%) with type 1 diabetes mellitus (patient-rated symptom response was excellent in 33, good in 25 and fair in 5 patients).[42] 4.1.3 Trials in Patients with Symptomatic Gastropathy Refractory to Other Agents The results of small, nonblind trials suggest that domperidone may also be effective in patients with symptomatic gastropathy unresponsive to other prokinetic and autiemetic agents. Domperidone 80 mg/day for 6 months reduced the mean symptom score from 17.8 (of a total possible 33) to 3.7 (79% decrease, p < 0.01) in 6 diabetic patients with nausea and vomiting who failed to improve with metoclopramide, prochlorperazine, chlorpromazine or trimethobenzamide.[41] A r Prospective trial in 57 patients with symptoms of diabetic gastropathy, who had previously discontinued metoclopramide because of either lack Adis International Limited. All rights reserved. 439 of efficacy or adverse events, also found that administration of domperidone 40 to 80 mg/day led to a markedly or moderately favourable response in 70% of patients, who successfully continued therapy with domperidone for a median of 377 days.[83] Similarly, domperidone 40 to 80 mg/day for up to 144 months significantly reduced the severity scores for nausea, vomiting, early satiety and eructation in 5 patients with type 1 diabetes mellitus whose symptoms of nausea and vomiting had not responded to 'standard' antiemetic therapy.[82] However, the severity of other symptoms such as regurgitation, heartburn, distension and flatulence did not improve with domperidone. The efficacy of domperidone was similar to that of cisapride (dosages not stated) in a nonblind trial in 55 diabetic patients with gastropathy unresponsive to metoclopramide; in 29 patients the reduction of symptom severity peaked at 12 months [a decrease in total symptom score from 9.3 (of total possible 15) to 3.9, 58% decrease] and some benefit was sustained in 13 evaluable patients after 24 months (total symptom score of 6.0, a 35% decrease from baseline). [84] The addition of cisapride 80 mg/day to domperidone 80 mg/day for a mean of 9 months also significantly improved the symptoms of gastropathy such as nausea, vomiting, satiety and bloating in 9 patients with type 1 diabetes mellitus who had not shown an adequate response with either agent alone; abdominal pain did not improve in these patients.[85] 4.2 Effects on Quality of Life Quality of life was assessed in patients with insulin-requiring diabetes mellitus with symptoms of gastropathy who underwent an 'enriched' trial[4,75] with domperidone (efficacy reported in table IV). Impairment of quality of life at baseline was demonstrated in 269 patients; scores on the SF-36 questionnaire were significantly lower than those in the general population. [86,87] Evaluable patients (in the single-blind enrichment phase), in whom the symptoms of gastropathy improved after domperidone 80 mg/day (n = 208), showed significant improve- Drugs 1998 Sep; 56 (3) Prakash & Wagstaff 440 ment in the physical and mental component summary scores of the SF-36 compared with those in whom symptoms did not respond to treatment (n = 61) after 4 weeks. In the second (double-blind) phase of the trial, responders to domperidone who were randomised to continue active treatment for a further 4 weeks (n = 104) continued to show improvement on the physical component summary score, while placebo recipients (n = 99) showed significant worsening. There was a significant difference in physical component summary scores between domperidone and placebo recipients at endpoint (+0.65 vs -1.77, p 0.05). The deterioration in mental component summary scores was similar in both groups.[86,87] Several smaller trials also indicate improvement in quality of life with domperidone treatment. The administration of domperidone (dosage not stated) for 12 months led to a significant symptomatic improvement (mean total symptom score decreased from 9.5 to 4.87, p < 0.01) in 7 patients with diabetic gastropathy in a nonblind trial and the quality-of-life status, as measured by the Sickness Impact Profile, improved in 6 (median improvement of 22%).[88] Similarly, 15 of 17 patients receiving domperidone 80 mg/day for up to 48 months reported physical domains of quality of life as good to excellent, and 13 rated their general health as better than before treatment.[44] istration of domperidone 40 to 120 mg/day for up to 79 months led to an increase in weight of 5 to 7kg, compared with pretreatment values, in small numbers of patients (n = 5 to 17 per trial) with symptoms of diabetic, idiopathic, sclerodermal or postsurgical gastropathy.[43,44,82] 5. Tolerability Although there are no published data from trials specifically designed to evaluate its tolerability, oral domperidone 40 to 120 mg/day administered from 1 month to 12 years was well tolerated in clinical trials involving patients with type 1 diabetes mellitus and gastropathy (section 4), In fact, the folerabilitv of domperidone 80 mg/day was similar to that of placebo in a 4-week, randomisec double-blind trial in 208 patients with type 1 diabetes mellitus and gastropathy.[89] The most common adverse events appear to be related to prolactin secretion. Breast symptoms, such as enlargement, soreness, secretion or tenderness, related to elevated prolactin levels, have been reported in 10 to 20% of patients.[43,77] However, breast symptoms occurred in 3 of 13 patients receiving domperidone and in a similar number of 4.3 Other End-points The effect of domperidone on the frequency of hospital admission of patients with symptoms of gastropathy has been evaluated in 5 small nonblind trials. Domperidone 40 to 120 mg/day for up to 8 years significantly decreased the hospital admission rate from pretreatment values in patients with gastropathy in 3 trials.[42-44] However, hospital admission rates remained unchanged in another trial (from 1.1 to 1.0 per patient per year)[84] when domperidone (dosage not stated) was administered for up to 36 months (fig. 3). The symptoms of gastropathy may be severe enough in some patients to cause nutritional deficiencies and weight loss.[ll] The nonblind admin- © Adis International Limited. All rights reserved, Fig. 3. Decrease in hospitalisation rates in patients with gastropathy receiving domperidone. Hospitalisation rates in patients with diabetic, idiopathic, scleroderma! or postsurgica! gastropathy receiving domperidone 40 to 120 mg/day in open-label trials of up to 3 years duration [42-44.84] * p < 0.05 vs baseline. Drugs 1998 Sep; 56 (3) Domperidone: A Review patients receiving placebo in a small double-blind, crossover trial.[76] These symptoms were mild to moderately severe and did not necessitate withdrawal of drug therapy. A retrospective analysis of data from 57 patients with diabetes mellitus with symptoms of gastropathy treated with domperidone 40 (n = 4) or 80 (n = 53) mg/day for up to 3 years (median of 246 days) reported adverse events in 22 patients.[83] Mild to moderate prolactin-related adverse events (such as galactorrhoea, breast tenderness/fullness and amenorrhoea) were observed n 16% of patients. Other adverse events reported by > 1 patient were gastrointestinal symptoms (11%), itching or rash (5%), muscle cramps (4%) and headache (4%)[83] (fig. 4). After 4 weeks of treatment, the severity of adverse events [scored on a scale from 0 (absent) to 3 (severe); total possible score 18] with domperidone 80 mg/day was significantly less than that with metoclopramide 40 nig/day (2.5 vs 4.5. p < 0.02) in 93 patients with type 1 diabetes mellitus in a randomised, double-blind, multicentre trial.[78] Domperidone recipients had a significantly lower incidence of somnolence and reduced mental acuity than those receiving metoclopramide.[78] Although CNS effects with oral domperidone arc rare in (he presence of an intact blood-brain barrier, acute dystonic reactions such as orolingual dyskinesia, oculogyric crisis and opisthotonus after oral domperidone 20 and 50mg have been described in 2 women aged 16 and 28 years with polycystic ovary syndrome.[90] The investigators suggest that hyperestrogenism in these patients may have increased the risk of acute dystonic reactions with domperidone. [90] Because intravenous administration of domperidone has been associated with an increased risk of cardiovascular events, such as cardiac arrest,[91,92] parenteral formulations of domperidone are no longer available for clinical use. 6. Dosage and Administration Domperidone is available as l0mg tablets, a 5 mg/ml oral suspension and 30mg suppositories.[93] © Adis International Limited. All rights reserved. 441 Fig. 4. Adverse events (AE) in patients receiving domperidone. Results of a retrospective study in 57 patients with type 1 diabetes mellitus with gastropathy who had discontinued metoclopramide because of adverse effects or lack of efficacy and who then received domperidone 40 to 80 mg/day for 47 to 377 days.[83] Prolactin-related AE included galactorrhoea, breast tenderness or fullness and amenorrhoea. Gastrointestinal (Gl) symptoms were not defined. The recommended dosages of domperidone in adults and in children are shown in table V. The most commonly used dosage of oral domperidone in clinical trials in patients with type 1 diabetes mellitus anda gastropathy (section 4) was 80 mg/day (range from 40 to 120 mg/day). Although domperidone is not recommended for long term use and a course of treatment with domperidone for the management of patients with nausea and vomiting should not exceed 12 weeks, [93] domperidone 40 to 80 mg/day has been used successfully for up to 8 to 12 years in long term trials in patients with type 1 diabetes mellitus with symptoms of gastropathy.[42,82] Domperidone may be used with caution in patients with renal impairment.1931 7. Place of Domperidone in the Management of Diabetic Gastropathy Symptoms of diabetic gastropathy are often chronic and difficult to treat. Achieving glycaemic control and modification of dietary intake such as ingestion of small frequent meals with low fat and fibre content may improve gastric emptying in some patients with diabetes mellitus.[11,94] However, the rationale for pharmacotherapy of diabetic Drugs 1998 Sep; 56 (3) 442 Table V. Recomme'nded dosage of domperidone for the management of nausea and vomiting due to various causes[93] patients with gastropathy is based on the occurrence of gastrointestinal symptoms, irrespective of delayed or normal gastric emptying.[94] Surgical procedures such as a venting gastrostomy for gastric decompression and a jejunostomy catheter for enteral feeding are reserved for patients with severe gastroparesis.[8,11] Drugs commonly used in the treatment of patients with diabetic gastropathy include metoclopramide, cisapride and domperidone. However, there is a lack of quality data comparing these drugs. The available data on the efficacy of domperidone in the management of patients with diabetic gastropathy have several limitations: few trials have been published in full, blood glucose levels were not monitored in included patients, none of the fully published trials compared the effects of domperidone with other gastrokinetic agents, and most trials were nonblind and conducted in small numbers of patients. Nonetheless, oral domperidone 40 to 80 mg/day significantly reduced the intensity and frequency of symptoms of gastropathy from baseline values in all available trials. The efficacy of domperidone in reducing the symptom severity of diabetic gastropathy was significantly greater than that of placebo and similar to that of metoclopramide 40 mg/day in double-blind trials and was similar to that of cisapride 40 mg/day in nonblind trials. In 'enriched' trials, domperidone led to improvement in the symptoms of 66 to 88% of patients in the initial © Adis International Limited. All rights reserved. Prakash & Wagstaff nonblind or single-blind phase. In the subsequent randomised single-blind or double-blind phase, the subgroup of patients with delayed gastric emptying who continued with domperidone showed significantly less deterioration of symptoms after 4 weeks than did those receiving placebo. There was a significant reduction in hospital admission rates in patients with gastropathy receiving domperidone. Quality of life also improved in patients whose symptoms responded with domperidone as compared with placebo recipients. The administration of domperidone has shown sustained symptomatic benefit in patients with diabetic gastropathy for up to 12 years. Diabetic patients with preserved autonomic function and those with delayed gastric emptying appear to respond more favourably to domperidone than those in whom autonomic function is damaged or those who have normal gastric emptying. In addition, domperidone may be effective in patients who are refractory to other agents and the combination of domperidone with gastrokinetic agents with a different mechanism of action, such as cisapride, may improve symptoms of patients who do not show an adequate response with domperidone alone. The tolerability profile of domperidone 80 mg/day is significantly superior to that of metoclopramide 40 mg/day. Metoclopramide has been associated with adverse CNS effects in 20% of recipients.[10] Although domperidone administration causes elevation of plasma prolactin levels, prolactin-related adverse events such as galactorrhoea, breast tenderness and fullness and amenorrhoea have been observed in only 16% of domperidone recipients. No serious adverse events have been reported with the use of domperidone. In conclusion, domperidone 40 to 80 mg/day appears to be an effective and well tolerated agent for the management of symptoms of gastropathy in patients with type 1 diabetes mellitus. 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Gastric myoelectrical activity in symptomatic diabetic patients with or without gastroparesis [abstract]. Gastroenterology 1995; 108 (4 Suppl.): A630 49. Koch KL, Bingaman S, Stern RM. Withdrawal study of domperidone vs placebo in diabetic patients: effect on upper gastrointestinal symptoms and gastric myoelectrical a c t i v i t y (abstract]. Gastroenterology 1995; 108 (4 Suppl.): A630 50. Bernini GP, Lucarini AR, Franchi F, et al. Humoral effects of ineioclopramide and domperidone in normal subjects and in hypertensive patients. J F.ndocrinol Invest 1988 Nov; M: 711-6 51. Brown GM, Verhaegan H, Van Wimersma Greidanus TB, et al. Endocrine effects of domperidone: a peripheral dopamine blocking agent. Clin Endocrinol Oxf 1981 Sep; 15: 275-82 52. Coiro V, Butturini U, Gnudi A, et al. TSH and PRL responses to domperidone and TRH in men with insulin-dependent diabetes mellitus of different duration. Horm Res 1987; 25: 206-14 53. Nerozzi D, Magnani A, Sforza V, et al. 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Scand J Gastroenterol 1995; 213 Suppl.: 7-16 Correspondence: Amitabh Prakash, Adis International Limited, 41 Centorian Drive, Private Bag 65901, Mairangi Bay, Auckland 10, New Zealand. E-mail: demail@adis.co.nz Drugs 1998 Sep; 56 (3)