Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

Study Overview
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Brief Summary
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This study is a longitudinal and cross-sectional evaluation of patients with Chronic Granulomatous Disease (CGD) who received or are receiving hematopoietic cell transplantation (HCT) for their disease under a variety of protocols used by participating institutions compared to a control non-HCT group receiving standard care. Investigators at multiple centers caring for patients with CGD in North America and 3 centers in Europe will participate. Patients with CGD will have been treated according to institutional practice and protocols. Investigators will enroll these patients as subjects in this protocol. This study will investigate which patients benefit most from HCT, and what types of transplants are optimal for patients with CGD, in the context of overall outcomes in CGD patients with and without transplant.

Detailed Description
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This study is a longitudinal and cross-sectional evaluation of patients with Chronic Granulomatous Disease (CGD) who received or are receiving hematopoietic cell transplantation (HCT) for their disease under a variety of protocols used by participating institutions compared to a control non-HCT group receiving standard care. Investigators at multiple centers caring for patients with CGD in North America and 3 centers in Europe will participate. Patients with CGD will have been treated according to institutional practice and protocols. Investigators will enroll these patients as subjects in this protocol. This study will investigate which patients benefit most from HCT, and what types of transplants are optimal for patients with CGD, in the context of overall outcomes in CGD patients with and without transplant.

Official Title
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Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

Conditions
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Chronic Granulomatous Disease

Participation Criteria
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Eligibility Criteria
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Both HCT and non-HCT subjects must be over the age of 2 and actively enrolled and receiving treatment under a CGD protocol at NIAID.

Ages Eligible for Study
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Minimum Age: 3 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cross Sectional<br>3 years after Transplant | |
| No Transplant<br>These patients were diagnosed with CGD but have not been transplanted | |
| Prospective<br>Prior to Transplant Conditioning | |
| Retrospective<br>1 year after Transplant | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The primary objective of this protocol is to estimate the 1- year, 2-year and 3-year (and longer if possible) overall survival probabilities post-HCT of CGD subjects born on or after 1988 who receive HCTon or after 1995. | Clinical outcomes (survival, infection, autoimmune disease, chimerism, GVHD) | 1, 2 or 3 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To compare overall survival from birth between patients born on or after 1988 who receive HCT on or after 1995 vs. those born on or after 1988 who receive conventional therapy, after adjusting for differences in year of birth and oxidase acti... | Clinical outcomes (survival, infection, autoimmune disease, chimerism, GVHD) | 3 years post Transplant or Non Transplant | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Rare Diseases, Primary Immune Deficiency Treatment Consortium, Retrospective, Natural History