Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

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Brief Title

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

Official Title

Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)

Brief Summary

      Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems
      both with the immune system and with easy bruising and bleeding. The immune abnormalities
      cause patients with WAS to be very susceptible to infections. Depending on the specific type
      of primary immune deficiency diseases, there are effective treatments, including antibiotics,
      cellular therapy and gene therapy, but studies of large numbers of patients are needed to
      determine the full range of causes, natural history, or the best methods of treatment for
      long term success.

      This multicenter study combines retrospective, prospective and cross-sectional analyses of
      the transplant experiences for patients with WAS who have already received HCT since 1990, or
      who will undergo Hematopoietic cell transplant (HCT) during the study period. The
      retrospective and prospective portions of the study will address the impact of a number of
      pre and post-transplant factors on post-transplant disease correction and ultimate benefit
      from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years
      post-HCT in consenting surviving patients.

Study Type


Primary Outcome

Longitudinal Analysis: Overall Survival From Time of HCT/Gene Therapy

Secondary Outcome

 Longitudinal Analysis: Proportion of Participants Achieving Hematologic Reconstitution


Wiskott-Aldrich Syndrome

Study Arms / Comparison Groups

 Retrospective Cohort (Longitudinal Analysis)
Description:  Participants with WAS treated at consortium centers since 1990 who have received transplant (Stratum A) or gene therapy (Stratum B)


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Estimated Enrollment


Start Date

February 2, 2014

Completion Date

May 1, 2019

Primary Completion Date

May 1, 2019

Eligibility Criteria

        Inclusion Criteria:

          -  WAS participants will be defined as males who have:

               1. thrombocytopenia (< 100K) AND EITHER molecular diagnosis of WAS OR reduced WASP
                  expression; OR

               2. thrombocytopenia (< 100K) AND positive family history consistent with WAS
                  diagnosis; OR

               3. chronic thrombocytopenia (< 100K for minimum of 3 months) AND low mean platelet
                  volume (MPV below normal range for age) AND EITHER recurrent and/or severe
                  infections requiring treatment and/or eczema OR lack of antibody response to
                  polysaccharide antigens or low IgM.

          -  Longitudinal Analysis (Retrospective and Prospective)

               1. Stratum A. Participants with WAS who have or will Receive HCT

                    -  Participants with WAS who have received an HCT since January 1, 1990

               2. Stratum B. Participants with WAS who have or will Receive Gene Transfer

                    -  Participants in which the intention is to treat with gene transfer with
                       autologous modified cells

          -  Cross-Sectional Analysis (Strata A and B) 1. Participants with WAS who are surviving
             and at least 2 years after the most recent HCT or gene therapy.

        Exclusion Criteria:

          -  As this is a natural history study, for both the Longitudinal Analysis and the
             Cross-Sectional Analysis we will not exclude any patients due to race or age who fit
             the inclusion criteria.




N/A - N/A

Accepts Healthy Volunteers



Lauri M. Burroughs, MD, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Responsible Party


Study Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)


 Primary Immune Deficiency Treatment Consortium (PIDTC)

Study Sponsor

Lauri M. Burroughs, MD, Study Chair, Fred Hutchinson Cancer Center

Verification Date

August 2020