Non-Muscle Invasive Bladder Urothelial Carcinoma St. Louis University Hospital Division of Urology Michael Mastromichalis, MD Outline Diagnosis & Epidemiology Staging Endoscopic Management Complications Repeat TURBT and Random Biopsies Immunotherapy Primary Intravesical Chemotherapy Radiation Surveillance Protocols Secondary Prevention Strategies Bladder Anatomy The urinary bladder has 3 distinct histologic layers Urothelium Lamina Propria Detrusor (Muscularis Propria) Bladder Urothelial Carcinoma Bladder Cancer Presentation Typical Symptoms Risk Stratification Grade & Stage CIS Multicentricity Lymphovascular Invasion Cytology and Molecular Markers Asymptomatic Patients / Autopsy Rates Urothelial Carcinoma UC represents over 90% of all bladder cancers diagnosed in the US 68,000 new cases are diagnosed per year >90% diagnosed are older than 55 13,000 deaths annually 500,000 survivors currently in the US 3:1 male to female, with incidence rising in all groups Lifetime risk of 1/28 Bladder Urothelial Carcinoma Smoking is the #1 risk factor Amines, 4-aminobiphenyl & analines are the culprits Aromatic amines in dyes, solvents, paints, combustion products, rubber, and textiles are also risk factors Hairdressers, mechanics, truckers Phenacetin derived analgesics Not coffee and artificial sweeteners Rarely familial syndrome with DNA mismatch repair (Lynch II) Slow acetylators (40% higher) vs fast acetylators Bladder Urothelial Carcinoma The vast majority of bladder UC are the result of environmental exposuretobacco Endogenous molecular factors play a role Cyclophosphamide & ifosfamide chemo A. fangchi herbs & arsenic Radiation therapy Prostate, anal, cervix Bladder Urothelial Carcinoma The entire urothelium is susceptible to carcinogenic insult and thus, to malignant transformation A “field change disease” Tumorgenesis separated by time and space Cells migrate and implant vs. multifocal carcinogenesis Urothelial Carcinoma The urinary bladder is the reservoir of urine and therefore has a prolonged “face-time” with renally excreted carcinogens UC has a long latency from exposure to cancer development supporting the theory of a carcinogenic cumulative effect on malignant transformation of the urothelium 48,000 Men over 10 years- UC incidence re: fluid intake 1.5L of water/day << less than 240mL Non-Muscle Invasive UC Historically known as ‘superficial’ bladder cancer Wide range- Low-grade papillary to high grade T1 with CIS 70-75% are amenable to bladder sparing treatments Grade 1,2,3 vs. Low/High Grade- regardless of invasion or CIS presence All tumors that have not invaded the detrusor Tumor Grading Ta denotes a papillary (LG or HG) tumor confined to the urothelium T1 is a papillary, sessile or nodular tumor invading the lamina propria (LG or HG) Anything beyond the urothelial basement membrane until the detrusor. Examples of Cystoscopic Tumor Examples of Cystoscopic Tumor Cystoscopy Cystoscopy Tumor Grading CIS is a flat, high-grade, tumor confined to the urothelium. No lamina propria invasion. Velvety, erythematous and easily missed on cystoscopy Severe atypia and nuclear aplasia with disorderly architecture Can be multicentric and often occur with high-grade tumors Ominous CIS undermining of adjacent healthy urothelium Non-Muscle Invasive Bladder Cancer Ta Urothelial Ca Low Grade Ta Urothelial Ca High Grade Carcinoma in Situ Definition and Epidemiology 75% of all urothelial bladder tumors are NMI Ta (70%) T1 (20% CIS (10%) Gross Hematuria- 15-35% risk of bladder tumor Microscopic Hematuria- 1-5% have bladder tumor New Irritative Symptoms- double the risk + CIS risk Non-Muscle Invasive Bladder Cancer Who progresses% and dies% from NMIBC? PUNLMP Papillary, LG Papillary, HG T1, HG CIS 3 5-10 15-40 30-50 >50 0-1 1-5 10-25 30 HG precursor Staging Non-Muscle Invasive Bladder Cancer Papillary Urothelial Neoplasm of Low Malignant Potential- PUNLMP Orderly cellular arrangement Minimal architectural abnormalities Minimal cellular atypia No cytologic features of malignancy, so unlikely to progress, ergo, they are considered benign Follow-up still recommended Tumor Biology & Behavior Invasive vs Non-invasive Urothelium- devoid of vessels or lymphatics Lamina propria- rich in both providing a suitable scaffold for metastasis and tumor dissemination Behavior (Progression) is primarily grade dependent HG has high recurrence and progression regardless of Ta or T1 status Tumor Biology & Behavior Low Grade vs. High Grade Disease Pathways These are essentially different diseases with different pathophysiology Bladder cancer, the early years... Tumor Biology & Behavior First hit- it all starts with altered cellular metabolism after exposure to detoxified or partially detoxified carcinogens Oxidative cellular DNA damage is the result Second hit- genetic or acquired cellular failure that promotes tumor, fails to inhibit tumor, or fails to repair oxidized/damaged DNA… – Activate oncogenes (RAS gene family) – Mutated tumor suppressor genes (Rb and P53) – Damaged APEX-1 Tumor Biology & Behavior Oxidative DNA damage causes chromosomal alterations Low Grade Pathway (Ta papillary tumors) More common Much fewer chromosomal mutations & abnormalities Usually indolent unless convert to high grade pathway Loss of part or all of Chromosome 9 (q) High Grade Pathway (CIS, T1, and muscle invasive) Numerous and variable chromosomal gains and losses Rb & P53 mutations, CH 7, 9, 17 (where p53 is located) Aggressivity: high p53, Ki-67, loss of E-Cadherin Low: No loss of E-Cadherin Tumor Biology & Behavior Most important risk factor in NMIUC progression is GRADE…then presence of CIS Ergo, High Grade Ta = High Risk Should be surveyed as such CIS=High Grade=High Risk and is a high grade, invasive urothelial cancer precursor Should be treated as such because 45-80% will progress to muscle invasive UC if untreated. Tumor Biology & Behavior NMIUC Prognosis correlates with: Tumor grade +/- CIS Tumor Size Multiplicity Papillary vs Sessile +/- Lymphovascular Invasion Tumor Biology & Behavior A Gentle Word re: HGT1 Lesions HGT1 tumors are usually papillary but are the most understaged tumors in bladder cancer 40% are understaged at time of cystectomy Only half of these are organ confined. Hydronephrosis usually indicates detrusor invasion 85-90% association with MI urothelial carcinoma Tumor Biology & Behavior Nodular or sessile appearance usually indicates deeper muscle invasion Micropapillary cancer is a very aggressive variant BCG and chemo resistant Early cystectomy for non-muscle invasive disease Tumor Biology & Behavior A Gentle Word re: HGT1 Lesions Some investigators have suggested a new grade if +deep lamina propria muscularis mucosae involvement LV invasion “T1b” Due to anecdotal risk of recurrence and progression. Endoscopic Management Office-based cystoscopy is the mainstay of diagnosis and surveillance. Entire urethra, prostate, bladder neck, and bladder Quality of efflux from each ureteral orifice Extent, location, number, and nature of tumors as well as UO proximity, mucosal irregularities or urethral involvement should be recorded and/or photographed. Urine cytology is encouraged for baseline and may encourage future random biopsies if positive Endoscopic Management TURBT is the initial treatment for visible lesions. Performed under regional or general anesthesia Need bimanual exam before prep and drape and after case for staging. Cytology with cystoscopy can be helpful as a baseline marker for future surveillance and treatment monitoring Endoscopic Management Resectoscope Examples Bladder Tumor Resection Examples Bladder Tumor Resection Endoscopic Management Retrograde pyelography if upper tract studies are insufficient or a positive radiographic finding Ipsilateral ureteral cytology, saline lavage, brush biopsy, or ureteroscopic resection for tissue Some advocate transurethral biopsy of the prostatic urethra for complete staging in men Endoscopic Management Essential to resect all of tumor ultimately to a depth of the detrusor for accurate staging Separating superficial and muscle swipes may aid the pathologist in identifying muscularis propria from muscularis mucosa An increase in abdominal fullness or girth requires a cystogram to r/o intraperitoneal perforation A cystogram is required prior to post-TURBT intravesical instillation Endoscopic Management Obturator Reflex Minimized with paralytic and avoiding overdistention Bipolar in saline minimizes as well Cystoscopic planning via 70 degree lens Resection via 30 degree lens Use of continuous flow with minimal fill Minimal filling to minimize bladder movement and detrusor thinning Endoscopic Management Conservative treatment of diverticular tumors – Should be sampled rather than resected – A minority advocate “purposeful perforation” – Partial cystectomy – Random biopsies would be warranted in preop planning TURBT should proceed without worry of the UO – Pure cut across UOs minimizes scarring – Stenting to manage oedema and healing Endoscopic Management Small, recurrent tumors- may be amenable to cold-cup biopsy (older women) and Bugbee electrode to tumor bed – Laser fulguration of the tumor bed in high risk patients is surgeon dependent Staged resections for bulky disease Endoscopic Management Complications Obturator reflex perforation Bleeding TUR Syndrome UO Obstruction Unrecognized disease Perforation Extraperitoneal Intraperitoneal Why Do Patients Recur? (and later, what can the urologist do about it) Nature of the tumor… Poor Protoplasm Missed tumors at TURBT Incomplete TURBT resection Implantation of shed tumor cells at TURBT A de novo tumor due to a tumor-sensitized, “atrisk” urothelium Field change disease and the urothelium will dedifferentiate at its leisure Endoscopic Management 2nd Look (Restage) TURBT When tumor volume, inaccessibility, and intraoperative medical instability warrant a second look for patient safety. Recommended 2-6 weeks after all HGTa & T1 tumors OR if no muscle present 40% positivity of re-staging sites of HG tumors and 20-50% likelihood of T-upgrade to MI disease Endoscopic Management Random Biopsies Is controversial. – – Some advocate when office cytology is + or to f/u with CIS treatment But denuded bladder suitable for tumor implanation Cold-cup utilized, Bugbee for hemostasis Not indicated in low-risk patients and those with negative cytology. Essential for preoperative planning in partial cystectomy or neobladder (urethral sparing) Frequently Asked… Concurrent TURP & TURBT with a hx of LG appropriate Concurrent TURP + TURBT with hx of HG or CIS should be staged In neobladder planning, prostate TUR biopsies are necessary but must be weighed against risk of high grade tumor seeding and possible dissemination MMC after TURBT Mitomycin C is the safest & most effective peri-TUR intravesical chemotherapeutic agent Single dose within 6 hours Intravesical “face-time” of 1 hour Recommended in those with low and high risk features Recurrence rate decreased by 30-50% and increased recurrence-free interval Destroys residual microscopic tumor at the TURBT site Used to prevent tumor implantation Initial tumors on the floor and side walls while recurrences at the dome MMC after TURBT If “healthy” or “academic” swipes taken, a cystogram can avert systemic complications from extravasated absorption by holding MMC Local irritative symptoms are common and more serious sequelae have occurred with perforation. Benefit seen from LGTa to HGT1, solitary papillary to multiple tumors (across the board) Staging CT urograms often accompany the patient after hematuria discovered Chest Roentgenogram Chest CT if pulmonary metastasis suspected clinically or an abnormal chest x-ray Comprehensive metabolic panel with hepatic components and alkaline phosphatase, CBC, & coagulation studies Elevated alk phos or bone pain = bone scan Intravesical Therapy Urologists should discuss treatment options and associated risks, side-effects, and benefits. A wide variety of agents, combinations, durations, and outcomes are reported. There is a true lack of uniformity regarding optimal doses, number of doses, and timing of instillations for inductions and maintenance therapies The optimal interval nor duration of cystoscopic follow up has been defined. “We’ve not done a great job with bladder cancer” M.J. Chehval, MD Intravesical Therapy Goal is to treat residual or unresected disease Prevent future recurrences and progression Delay the need for more aggressive surgical intervention Prevent tumor implantation Intravesical Therapy Takes advantage of the relatively low absorptivecapacity of bladder Noninvasive access to cancer site Relative avoidance of systemic exposure to chemotherapy Innovator and Pioneer of Intravesical Immunotherapy Immunotherapy Goal of immunotherapy is to Augment cancer cell recognition Promote tumor cell-specific cytotoxicity Recruit tumor cells that have evaded the immune system “onto the radar” If “revved” up, the BCG immune response can mimic tumor stimulated, tumor specific cytotoxicity for years Immunotherapy BCG Bacillus Calmette-Guerin Live, attenuated Mycobacterium bovis Developed by Albert Calmette and Camille Guerin at the Pasteur Institute • Used initially as a Tb vaccine Massive local immune response all reflecting a Th1 process driven by… Direct binding of fibronectin within the bladder wall Immunotherapy BCG Use in CIS CIS is often diffuse preventing complete tumor resection 80% response rate 50% durable at 4 yrs and 30% at 10 yrs Higher efficacy compared with intravesical chemo Induction vs. induction + maintenance Immunotherapy BCG Use in residual tumor Effectively treats Ta papillary lesions, but not a surgical substitute TURP + delayed BCG to prostatic urethra is effective treatment for prostatic CIS Use as prophylaxis for 6 weeks after TURBT Induction* decreased recurrence by up to 40% for T1 lesions compared to TUR alone Induction* + Maintenance* can reduce progression by 2030% in HG tumors Maintenance is thought to provide long-term immunostimulation BCG Scheduling 6 week induction alone is insufficient to achieve optimal response Lamm and SWOG Maintenance – (after 6 week induction) @ 3 months- 3 weekly instillations @ 6 months- 3 weekly instillations then every 6 months for 3 years 18 more instillations BCG Scheduling SWOG scheduling had a high dropout rate due to side effects Most consider 1 year of maintenance to be adequate Lengthening interval and decreasing the dose can help with bothersome symptoms Multifocal, CIS & HG tumors are where benefit seen Contraindications Absolute Immunosuppressed and immunocompromised Immediately after TURBT/TURP, gross hematuria or traumatic foley (disrupted urothelium) Hx of BCG Sepsis Relative Active UTI Total incontinence Liver disease Hx of TB Poor performance status or advanced age BCG Toxicity Treatments Moderate Irritative Symptoms, hematuria, afebrile (<48hrs) Get urine culture Anticholinergics, pyridium, analgesics & NSAIDS Severe Irritative Symptoms, Fevers, or >48hrs Urine Culture, CXR, LFT’s ID Consult Isoniazid and rifampin until symptoms resolve Dose reduction when instillations resume BCG Toxicity Treatments Serious Complications Hemodynamic changes (BCG Sepsis), highgrade fevers, allergic reactions, solid organ involvement with fevers & rigors – Blood and Urine Cultures, CXR, LFT’s – Steroids, antihistamines, broad-spectrum antibiotics – ID Consult Isoniazid, rifampin, ethambutol, for 3-6 months Immunotherapy Interferon-2b/Interferon-α Mechanism is via lymphocyte activation, cytokine release Less effective than BCG or intravesical chemo Not effective at eradicating residual disease, preventing recurrence, or treating CIS inherent antiproliferative and antiangiogenic properties Has been studied as an adjunct to BCG in an effort to lower BCG dose Failed to demonstrate equivalence. Garlic and Mistletoe extracts as immunogenics University of Chicago BCG Treatment and Surveillance Protocol for ≥HGTa Initial TURBT After 4 weeks, Re-TURBT (bc HG Ta and all T1 disease) *After 6 weeks, BCG x 6 weeks (induction) Cystoscopy surveillance at 3 month mark* 3 Weeks of BCG Cystoscopy surveillance at 6 month mark* 3 Weeks of BCG Cystoscopy surveillance at 9 month mark* 3 Weeks of BCG Cystoscopy surveillance at 12 month mark* *from 1st dose of BCG induction All in all, 1 year's worth of cancer treatment induction + maintenance + 4 surveillance cystoscopies Intravesical Chemotherapy Mitomycin C An antibiotic derivative that inhibits DNA synthesis via alkylation A “larger” molecule • systemic absorption rare unless perforation Reduces recurrence and progression, although inferior to BCG induction & maintenance Attractive due to much less toxic than BCG 20-40mg/20-40mL of sterile water Palmar Desquamation MMC Chemical Cystitis Intravesical Chemotherapy MMC Some advocate dehydration + oral sodium-bicarbinate days prior to administration to reduce urinary degradation and increase efficacy Side effects- most common site Skin rash- palmar desquamation Irritative symptoms and chemical cystitis (10%) Rarely, contracted bladder MMC I + M proved superior to MMC Induction alone In both recurrence and progression MMC I + M proved inferior to BCG I + M in all comers Intravesical Chemotherapy Doxorubicin, Valrubicin & Epirubicin Doxorubicin Inhibits topoisomerase II and thus inhibits protein synthesis Shown to prevent recurrence but not progression Valrubicin Approved for treatment of BCG refractory CIS who refuse or are unfit for radical cystectomy 20% complete response Epirubicin Decreases recurrence when compared to TUR alone Not FDA approved in US Intravesical Chemotherapy Thiotepa & Others… Only agent approved for treatment of papillary urothelial bladder cancer Alkylating agent that is >50% absorbed The original and cheapest intravesical agent Myelosuppression Gemcitabine & docetaxel intravesically currently being investigated Radiation Therapy • Has not been studied extensively in NMI Urothelial Ca • Initial very good response, short term • Not effective long term for Ta or CIS – 90% recur in 5 years Clinical Follow-Up Patient History and GU Physical U/A Cystoscopy esp 1st 3mo post-TURBT cystoscopy Urine Cytology Urinary Markers? Upper tract imaging… Early Cystectomy • Should be considered in patients who Micropapillary Variant! – Do not tolerate intravesical therapy – Failed attempts at disease control with TURBT +IVT – Lesions not amenable to endoscopic resection – Failure of TURBT and intravesical therapy • • • • Recurrence at higher grade and multifocality Progression on intravesical therapy (Grade Progression) Invasion into detrusor (T progression) Especially in HGTa or CIS Extravesical Imaging Surveillance Most patients undergo upper tract imaging for initial hematuria workup High grade or multiple tumors warrant upper tract annual imaging surveillance every 1-2 years Changes in cytology warrant investigation If upper tract disease is discovered, mortality rate jumps to ~50% for all-comers Must individualize based on patient’s risk of recurrence and progression to extravesical sites. Upper tract surveillance for low risk disease not required Upper tract recurrence <0.9% in low grade Ta disease Secondary Prevention Strategies Goal is to reduce the risk of recurrence and progression Minimize exposure to carcinogens and smoking Increased fluid intake Reduces concentration and dwell time of carcinogens Low-fat, low cholesterol diet Vitamin A and B6 have been disappointing High Dose MTV- advantage seen at 5 years Oncovite championed by Don Lamm, MD Suppresses partially transformed cells Hepatotoxic >40K IU per day Urine Cytology Voided/Cystoscopically attained urine specimen is examined for exfoliated cancer cells Less effective for LG tumors (30% sensitivity) Well differentiated and normal cells retain their cohesive properties and are less commonly shed Sensitivity and specificity are quite high for HG and CIS, although subjective (pathologist) Positive test is not an indication for treatment but does warrant upper and lower tract workup + TUR prostate strip Urine Cytology Can be used to screen, evaluate, & follow-up high risk patients Can be used to monitor recurrence, progression and response to intravesical therapies Gravity vs. barbotage specimens Inform cytopathologist if specimen from bowel Urine Markers May aid in diagnosis and surveillance of patients with NMIUC Many commercially available NMP-22 BTA TRAK ImmunoCyt Urovysion FISH Under Investigation Although radical cystectomy is beyond the scope of this talk…. There is data re: early cystectomy in… high-risk, recurring and progressing patients those recurring at 3month post-TURBT cystoscopy Intravesical failures HGT1 tumors are usually papillary but are the most understaged tumors in bladder cancer 40% are understaged at time of cystectomy Only half of these are organ confined at time of cystectomy Long-Term Investigation Laser ablation therapy for known low-grade papillary tumors Argon, KTP, Holmium, & Neodynium-YAG In select lower and upper tract tumors with close surveillance No obturator nerve stimulation Not appropriate for new lesions or initial TURBT Collateral damage Office Fulguration In low risk and recurrent LGTa papillary tumors or papillomas Future Fluorescent Cystoscopy 5-aminolevulinic acid (5-ALA) A precursor to heme biosynthesis is instilled into the bladder Taken up by neoplasms Blue light excites the agent and can detect otherwise unseen CIS on white light Many false + due to inflammatory lesions Fluorescent Cystoscopy Fluorescent Cystoscopy Future Photodynamic Therapy Reactive oxygen species have an antitumor effect Activates a photosensitizing agent in the urothelium delivered systemically or intravesically Porfimer sodium 5-ALA In addition to irritative symptoms, tissue sloughing, bladder contracture, and VUR are well known side effects Future Molecular markers are being studied to predict recurrence, progression, and response to therapy Flow cytometry p53 and Rb in serum & urine Proliferative indices Urinary growth factors Matrix metalloproteins (MMPs) Urinary Plasminogen Activator El Fin References available upon request. Questions?