Download 1 - European Society for Immunodeficiencies

Indication and outcome of hematopoietic stem cell transplantation in primary
immunodeficiency with predominant hypogammaglobulinemia – a retrospective study
Objective:
The proposed study aims at evaluating patient numbers, outcome and immune reconstitution
after hematopoietic stem cell transplantation (HSCT) in patients with primary
immunodeficiency with predominant hypogammaglobulinemia / antibody deficiency (PAD) in
a retrospective multi-centric survey.
PAD is the most common inborn immunodeficiency in adults. Patients suffer from severe,
recurrent infections that can be associated with other co-morbidities such as autoimmunity,
granuloma, lymphoproliferation and malignancies. The most common malignancy in adult
PAD is lymphoma occurring in ~4-8% of patients. Hematopoietic stem cell transplantation
(HSCT) has been used for single PAD patients with secondary malignancy or suspected
combined immunodeficiency, however, its risks and benefits for PAD patients and the
circumstances under which HSCT can cure the immunodeficiency have not been assessed
systematically.
Eligibility: All available patients (≥ 2 years at transplantation) with primary immunodeficiency
with predominant antibody deficiency having undergone HSCT for any reason until March
2013. Predominant antibody deficiency is defined as severe reduction in at least 2 serum
immunoglobulin isotypes with normal or low number of B cells (IUIS criteria table 3.2) or as a
a severe reduction in all serum immunoglobulin isotypes with profoundly decreased or
absent B cells (IUIS criteria table 3.1). The requested information is part of the dataset of the
EBMT (European Group for Blood and Marrow Transplantation) and the ESID (European
Society for Immunodeficiencies) registries. Patients have to consent to either data collection
via ESID or EBMT registry.
Endpoints: In this study we will combine transplant related data and data specific for
immunodeficient patients (major infection frequency, lymphocyte counts, vaccination
response, immunoglobulin levels pre- and post-transplant) in order to assess number,
indication, mortality, morbidity and immune reconstitution after HSCT in PAD patients. Adult
and pediatric patients will be compared with regard to reconstitution of the adaptive immune
system.
Duration of the data collection period: 3-6 months
Contact:
Prof. Dr. Klaus Warnatz
Prof. Dr. Jürgen Finke
Dr. Claudia Wehr
Dr. Marta Rizzi
[email protected]
[email protected]
[email protected]
[email protected]
Centre for Chronic Immunodeficiency and Department of Hematology and Oncolgy
University Medical Centre Freiburg
Breisacher Straße 117 – 2nd floor
79106 Freiburg
Germany
Tel: +49 (0)761 270-77640
Fax: +49 (0)761 270-77600