Research Proposal Form
Application for access to data collected in the UKPID online database
Dr Peter Arkwright
Applicant:
Frequency, distribution and clinical complications of
complement immunodeficiencies (excluding HAE) in Europe
Complement immunodeficiencies (excluding HAE)
Title of study:
Condition:
Current evidence
need for a study:
and
Complement immunodeficiencies are a rare group of PID
disorders. Excluding HAE, these patients are at increased risk
of both fulminant bacterial infections as well as in some
conditions autoimmune disease. Even specialist immunology
centres tend to see only a handful of patients with these
diseases. This European survey will provide an overview of the
frequency and distribution of complement immunodeficiencies
(excluding HAE, which has recently been reviewed) and the
complications and treatments currently provided to these
patients.
Objective(s):

Primary

Secondary
Determine the frequency and types of complement
immunodeficiencies (excluding HAE) in Europe
Analyse the morbidity (infection frequency and severity) and
mortality in relation to treatment (antibiotic use).
In/exclusion criteria:

Inclusion

Exclusion
Planned study duration:
Children and adults with complement immunodeficiencies
(excluding HAE) identified using the ESID database.
Patients not fitting the above criteria.
Patients in whom clinical and laboratory data is inaccessible.
November 2013 to November 2015
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It is important that the study concept is discussed with a statistician prior to initiating data extraction and
analysis.
Please add the following information:
1. Previous experience in research in primary immunodeficiencies (list 3 relevant
publications)
I am accredited by the GMC/RCPCH as a specialist in paediatric immunologist and have
worked as a specialist leading the paediatric immunodeficiency service for Manchester
and the North West of England for the last 11 years. I am also a senior lecturer in
paediatric immunology at the University of Manchester and have over 100 peer-reviewed
publications, many of which relate to PID. I am the lead investigator for Manchester for
the ESID registry.
A. Edgar JD, Buckland M, Guzman D, Conlon NP, Knerr V, Bangs C, Reiser V, Panahloo Z,
Workman S, Slatter M, Gennery AR, Davies EG, Allwood Z, Arkwright PD, Helbert M,
Longhurst HJ, Grigoriadou S, Devlin LA, Huissoon A, Krishna MT, Hackett S, Kumararatne
DS, Condliffe AM, Baxendale H, Henderson K, Bethune C, Symons C, Wood P, Ford K, Patel
S, Jain R, Jolles S, El-Shanawany T, Alachkar H, Herwadkar A, Sargur R, Shrimpton A,
Hayman G, Abuzakouk M, Spickett G, Darroch CJ, Paulus S, Marshall SE, McDermott EM,
Heath PT, Herriot R, Noorani S, Turner M, Khan S, Grimbacher B. The United Kingdom
Primary Immune Deficiency (UKPID) Registry: Report of the first 4 years’ activity 20082012. Clin Exp Immunol 2013; Jul 11.
B. Crawford G, Enders A, Gileadi U, Stankovic S, Zhang Q, Lambe T, Crockford TL, Lockstone
HE, Freeman A, Arkwright PD, Smart JM, Ma CS, Tangye SG, Goodnow CC, Cerundolo V,
Godfrey DI, Su HC, Randall KL, Cornall RC. DOCK8 is critical for the survival and function
of NKT cells. Blood 2013122:2052-61.
C. Jolles S, Williams P, Carne E, Mian H, Huissoon A, Wong G, Hackett S, Lortan J, Platts V,
Longhurst H, Grigoriadou S, Dempster J, Deacock S, Kahn S, Darroch J, Simon C, Thomas
M, Pavaladurai V, Alachkar H, Herwadker A, Abinun M, Arkwright P, Tarzi M, Helbert M,
Bangs C, Pastacaldi C, Phillips C, Bennett H, El-Shanawany T. A UK National Audit of
Hereditary and Acquired Angioedema. Clin Exp Immunol 2013 Jun 21.
2. A disclosure statement regarding potential conflicts of interest (such as financial
affiliations with pharmaceutical companies).
I declare no conflict of interests, financial or otherwise.
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Evaluation criteria for registry research proposals (1 - 5 points each):
 Is the proposal within the scope of the UKPID Database?
 Does this proposal address an important problem?
 Are the conceptual or clinical framework, design, methods, and analyses adequately
developed, and appropriate to the aims of the proposal? Is the study feasible?
 Is the proposal original and innovative?
 Are the investigators appropriately trained and suited to carry out this work? Does the
scientific environment in which the work will be done contribute to the probability of
success? One of the investigators should be a recognized specialist in the subject.
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