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

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

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 Overview

• Status: Completed
• Conditions: Wiskott-Aldrich Syndrome

• Study Type: Observational
• Enrollment (Actual): 305

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T3B 6A8
      • Alberta Children's Hospital
  • British Columbia
    • Vancouver, British Columbia, Canada, V6H 3V4
      • Children's & Women's Health Centre of British Columbia
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3E 0V9
      • Cancer Care Manitoba, University of Manitoba
  • Ontario
    • Toronto, Ontario, Canada, M5G 1XB
      • The Hospital for Sick Children
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • CHU Sainte-Justine, Department of Pediatrics, University of Montreal
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Department of Pediatrics, University of Alabama at Birmingham
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Children's Hospital
  • California
    • Los Angeles, California, United States, 90027
      • Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California
    • Los Angeles, California, United States, 90095-1752
      • Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles,
    • Palo Alto, California, United States, 94304
      • Lucile Salter Packard Children's Hospital at Stanford
    • San Francisco, California, United States, 94143-1278
      • University of California, San Francisco Benioff Children's Hospital
  • Colorado
    • Denver, Colorado, United States, 80220
      • Children's Hospital Denver, University of Colorado
  • Delaware
    • Wilmington, Delaware, United States, 19803
      • Nemours Alfred I. Dupont Hospital for Children
  • District of Columbia
    • Washington, District of Columbia, United States, 20010-2970
      • Children's National Hospital-George Washington University School of Medicine and Health Sciences
  • Florida
    • Saint Petersburg, Florida, United States, 33701
      • Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Aflac Cancer and Blood Disorders Center, Emory/Children's Healthcare of Atlanta
  • Illinois
    • Chicago, Illinois, United States, 60614
      • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Louisiana
    • New Orleans, Louisiana, United States, 70118
      • Center for Cancer and Blood Disorders, Children's Hospital/Louisiana State University
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • Department of Pediatrics, C. S. Mott Children's Hospital, University of Michigan
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Division of Pediatric Blood and Marrow Transplantation, University of Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic Children's Center
  • Missouri
    • Saint Louis, Missouri, United States, 63104
      • Cardinal Glennon Children's Hospital, Saint Louis University
    • Saint Louis, Missouri, United States, 63110
      • Saint Louis Children's Hospital, Washington University
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Institute for Pediatric Cancer and Blood Disorders, Hackensack University Medical Center
  • New York
    • New York, New York, United States, 10065
      • Department of Pediatrics, Memorial Sloan Kettering Cancer Center
    • Rochester, New York, United States, 14642
      • Department of Pediatrics, Golisano Children's Hospital, University of Rochester
    • Valhalla, New York, United States, 10595
      • Maria Fareri Children's Hospital, New York Medical College
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229-3039
      • Cincinnati Children's Hospital Medical Center, University of Cincinnati
    • Cleveland, Ohio, United States, 44106
      • Rainbow Babies and Children's Hospital
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Oregon
    • Portland, Oregon, United States, 97239
      • Division of Pediatric Hematology/Oncology, Oregon Health and Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia, University of Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • University of Pittsburgh Children's Hospital of Pittsburgh
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • St. Jude Children's Research Hospital
  • Texas
    • Dallas, Texas, United States, 75390-9263
      • Pediatrics, University of Texas Southwestern Medical Center
    • Houston, Texas, United States, 77030-2399
      • Baylor College of Medicine Section of Immunology, Allergy, and Retrovirology, Texas Children's Hospital
    • San Antonio, Texas, United States, 78229
      • Texas Transplant Institute, Methodist Children's Hospital
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Hospital, University of Utah
  • Washington
    • Seattle, Washington, United States, 98109
      • Fred Hutchinson Cancer Research Center and University of Washington-Seattle Children's Hospital
  • Wisconsin
    • Madison, Wisconsin, United States, 53705-2275
      • American Family Children's Hospital, University of Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Children's Hospital of Wisconsin-Milwaukee

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Male WAS participants treated at consortium centers Since 1990

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort
Retrospective Cohort (Longitudinal Analysis); Participants with WAS treated at consortium centers since 1990 who have received transplant (Stratum A) or gene therapy (Stratum B)
Prospective Cohort (Longitudinal Analysis); Participants treated at consortium centers since 1990 who will receive transplant (Stratum A) or gene therapy (Stratum B)
Cross-Sectional Cohort (Cross-sectional Analysis); Living participants with WAS who have received transplant (Stratum A) or gene therapy (Stratum B) at Consortium Centers 1990-Present And >= 2 Years Post-Transplant

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Longitudinal Analysis: Overall Survival From Time of HCT/Gene Therapy	The event analyzed is death from any cause. The time to this event is the time from HCT/gene therapy to death or last follow-up.	an expected average of 5 years
Cross-Sectional Analysis: Proportion of Participants Achieving Full T Cell Reconstitution	Full T cell reconstitution is defined by all of the following: CD3 cell count within normal range for age.; CD4 cell count within normal range for age.; CD8 cell count within normal range for age; Donor T cell chimerism > 95%; Lymphocyte proliferation to PHA. Proliferative responses to PHA within the normal range (at or above the lower limit of normal). When possible the proliferation to PHA should be calculated as a percentage of the lower limit of normal controls for that laboratory; alternatively, the lower limit of normal controls for the day may be used	an expected average of 5 years
Cross-Sectional Analysis: Proportion of Participants Achieving Full B Cell Reconstitution	Full B cell reconstitution is defined by all of the following: Quantitative immunoglobulins (IgG, IgM, and IgA) within normal range; each immunoglobulin level will be assessed separately.; Serologic confirmation of post immunization tetanus titer in protective range.; Serologic confirmation of post immunization pneumococcal titers in protective range (protective titers for > 50% of the serotypes contained in the vaccine) following immunization	an expected average of 5 years
Cross-Sectional Analysis: Proportion of Participants Achieving Resolution of thrombocytopenia	Resolution of thrombocytopenia defined by Platelets ≥ 150,000/microliter (transfusion independent for at least 7 consecutive days)	an expected average of 5 years
Cross-Sectional Analysis: Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL	Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL is defined as the first day of at least 3 different days the ANC is measured as 500 / uL or greater	an expected average of 5 years
Cross-sectional Analysis: Day of Recovery of Platelet Count to 20,000 / uL	Day of Recovery of Platelet Count to 20,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 20,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.	an expected average of 5 years
Cross-sectional Analysis: Day of Recovery of Platelet Count to 50,000 / uL	Day of Recovery of Platelet Count to 50,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 50,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.	an expected average of 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Longitudinal Analysis: Proportion of Participants Achieving Hematologic Reconstitution	Hematologic Reconstitution is defined as attainment of each of the following lab test values: Hemoglobin within normal range for age; WBC count within normal range for age; Absolute neutrophil count (ANC) within normal range for age; Platelet count ≥ 150,000/microL and without transfusion for at least 7 consecutive days	an expected average of 5 years
Longitudinal Analysis: Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL	Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL is defined as the first day of at least 3 different days the ANC is measured as 500 / uL or greater.	an expected average of 5 years
Longitudinal Analysis: Day of Recovery of Platelet Count to 20,000 / uL	Day of Recovery of Platelet Count to 20,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 20,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.	an expected average of 5 years
Longitudinal Analysis: Day of Recovery of Platelet Count to 50,000 / uL	Day of Recovery of Platelet Count to 50,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 50,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.	an expected average of 5 years
Longitudinal Analysis: Proportion of Participants Achieving Full T Cell Immune Reconstitution	Absolute CD3 T cell count within normal range for age; Absolute CD4 T cell count within normal range for age; Absolute CD8 T cell count within normal range for age; Proliferative responses to PHA within the normal range (at or above the lower limit of normal).; When possible the proliferation to PHA should be calculated as a percentage of the lower limit of normal controls for that laboratory; alternatively, the lower limit of normal controls for the day may be used.	an expected average of 5 years
Longitudinal Analysis: Proportion of Participants Achieving Full B Cell Immune Reconstitution	Quantitative immunoglobulins (IgG, IgM, and IgA) within normal range for age; each immunoglobulin level will be assessed separately.; Serologic confirmation of post immunization tetanus titer in protective range.; Serologic confirmation of post immunization pneumococcal titers in protective range (protective titers for > 50% of the serotypes contained in the vaccine) following immunization.; Patients who remain on IVIG will be considered not B cell reconstituted.; Normalization of isohemagglutinin titers.	an expected average of 5 years
Longitudinal Analysis: State of Lineage Specific Chimerism (HCT Stratum)	Peripheral blood chimerism will be assessed by FISH XX/XY, STRs, or WASP expression.	an expected average of 5 years
Longitudinal Analysis: Definition of Graft Failure / Rejection	Less than 5% of donor cells in all lineages or in whole blood by 100 days post-HCT using standard PCR based or in situ hybridization techniques OR; Second transplant by 100 days post-HCT (unless > 5% CD3 and purpose is to boost immune recovery). Failure to achieve ≥5% donor cells in all lineages or in whole blood by 100 days post-HCT will be defined as graft failure/rejection. Patients who receive a second transplant by day 100 will be considered graft failure.	an expected average of 5 years
Longitudinal Analysis: Severe bleeding episodes	Any severe bleeding episode requiring platelet and/or RBC transfusion(s)	an expected average of 5 years
Longitudinal Analysis: Malignancy	New onset or relapse of lymphoid malignancy confirmed by relevant pathologic and genetic features.	an expected average of 5 years
Longitudinal Analysis: Growth	Z score of weight and height pre-HCT/gene therapy and post-HCT/gene therapy.	an expected average of 5 years
Longitudinal Analysis: Incidence of Acute GVHD	The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the Consensus criteria of Grades IIIV or grades III-IV acute GVHD1 are considered events. Death is a competing risk, and patients alive without acute GVHD will be censored at the time of last follow-up.	an expected average of 5 years
Longitudinal Analysis: Incidence of Chronic GVHD	Chronic GVHD will be graded as limited or extensive.2 Occurrence of symptoms in any organ system that meet the criteria of chronic GVHD will be recorded. Death is a competing risk, and patients alive without chronic GVHD will be censored at time of last follow-up.	an expected average of 5 years
Longitudinal Analysis: Autoimmunity disorders	Incidence of documented autoimmunity disorders	an expected average of 5 years
Longitudinal Analysis: Infections / blood borne infections	Clinical resolution of any pre-HCT opportunistic infections including but not limited to CMV, HSV1, adenovirus, EBV, and VZV. Approximate time to resolution (clinically well, off treatment, and/or negative culture/PCR assay) will be measured from the day of HCT.; Incidence of documented severe (requiring hospitalization or resulting in death) and/or recurrent bacterial, viral or fungal infection post HCT. These will be reported by site of disease, organism, date of onset post HCT, and whether or not the infection resolved.; Presence and resolution of severe warts (verruca vulgaris, flat warts) from the day of HCT. Whether the subject had complete resolution, partial resolution, persistent or recurrent warts will be recorded.; New episodes of infections due to meningococcus, pneumococcus or hemophilus.; Lymphoproliferative disease due to EBV.	an expected average of 5 years
Cross-sectional Analysis: Current State of Lineage Specific Chimerism (HCT Stratum)	Peripheral blood donor chimerism will be assessed by FISH XX/XY, STR, and/or WASP expression	an expected average of 5 years
Cross-sectional Analysis: Current frequency and severity of infections		an expected average of 5 years
Cross-sectional Analysis: Current Status of Growth	Current Z score of weight and height pre-HCT/gene therapy and post-HCT/gene therapy.	an expected average of 5 years
Cross-Sectional Analysis: Graft-versus-host Disease (GvHD)	Presence of chronic GVHD, current assessment; graded as limited or extensive	an expected average of 5 years
Cross-Sectional Analysis: Autoimmunity Disorders	Presence of autoimmunity disorders	an expected average of 5 years
Cross-sectional Analysis: Severe Bleeding Episodes	Any severe bleeding episode requiring platelet and/or RBC transfusion(s) during the previous year.	an expected average of 5 years
Cross-sectional Analysis: fertility	Whether the subject has biological offspring will be recorded.	an expected average of 5 years
Cross-sectional Analysis: malignancy	New onset or relapse of lymphoid malignancy confirmed by relevant pathologic and genetic features.	an expected average of 5 years
Cross-sectional Analysis: Quality of Life Questionnaire	Age appropriate testing will be performed at the cross-sectional visit inpatients surviving at least two years post-transplant	an expected average of 5 years

Collaborators and Investigators

• Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Collaborators: Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment Consortium (PIDTC)
• Investigators: Study Chair: Lauri M. Burroughs, MD, Fred Hutchinson Cancer Center; Study Chair: David J. Rawlings, MD, Department of Pediatrics, University of Washington-Seattle Children's Hospital; Study Chair: Luigi D. Notarangelo, MD, National Institute of Allergy and Infectious Diseases, NIH; Study Chair: Alexandra H. Filipovich, MD, Children's Hospital Medical Center, Cincinnati

Publications and helpful links

General Publications

• Griffith LM, Cowan MJ, Kohn DB, Notarangelo LD, Puck JM, Schultz KR, Buckley RH, Eapen M, Kamani NR, O'Reilly RJ, Parkman R, Roifman CM, Sullivan KE, Filipovich AH, Fleisher TA, Shearer WT. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J Allergy Clin Immunol. 2008 Dec;122(6):1087-96. doi: 10.1016/j.jaci.2008.09.045. Epub 2008 Nov 6.
• Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC; workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014 Feb;133(2):335-47. doi: 10.1016/j.jaci.2013.07.052. Epub 2013 Oct 15.
• Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Shearer WT, Burroughs LM, Torgerson TR, Decaluwe H, Haddad E; workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) update. J Allergy Clin Immunol. 2016 Aug;138(2):375-85. doi: 10.1016/j.jaci.2016.01.051. Epub 2016 Apr 22.
• Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31;371(5):434-46. doi: 10.1056/NEJMoa1401177.
• Haddad E, Allakhverdi Z, Griffith LM, Cowan MJ, Notarangelo LD. Survey on retransplantation criteria for patients with severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Feb;133(2):597-9. doi: 10.1016/j.jaci.2013.10.022. Epub 2013 Dec 10. No abstract available.
• Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol. 2014 Apr;133(4):1092-8. doi: 10.1016/j.jaci.2013.09.044. Epub 2013 Nov 28.
• Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Shearer WT, O'Reilly RJ, Fleisher TA, Pai SY, Hanson IC, Pulsipher MA, Fuleihan R, Filipovich A, Goldman F, Kapoor N, Small T, Smith A, Chan KW, Cuvelier G, Heimall J, Knutsen A, Loechelt B, Moore T, Buckley RH. The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol. 2013 Oct;33(7):1156-64. doi: 10.1007/s10875-013-9917-y. Epub 2013 Jul 2.
• Griffith LM, Cowan MJ, Notarangelo LD, Puck JM, Buckley RH, Candotti F, Conley ME, Fleisher TA, Gaspar HB, Kohn DB, Ochs HD, O'Reilly RJ, Rizzo JD, Roifman CM, Small TN, Shearer WT; Workshop Participants. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. J Allergy Clin Immunol. 2009 Dec;124(6):1152-60.e12. doi: 10.1016/j.jaci.2009.10.022.
• Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing JJ, Edwards S, Dvorak CC, Chaudhury S, Prockop SE, Quinones R, Goldman FD, Quigg TC, Chandrakasan S, Smith AR, Parikh S, Davila Saldana BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez C, Chellapandian D, Shereck E, Miller HK, Kapoor N, Barnum JL, Chong H, Shyr DC, Chen K, Abu-Arja R, Shah AJ, Weinacht KG, Moore TB, Joshi A, DeSantes KB, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson T, Pulsipher MA, Haddad E, Sullivan KE, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings DJ, Cowan MJ. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood. 2020 Jun 4;135(23):2094-2105. doi: 10.1182/blood.2019002939.

Helpful Links

• National Institute of Allergy and Infectious Diseases (NIAID)
• Division of Allergy, Immunology, and Transplantation (DAIT)
• Primary Immune Deficiency Treatment Consortium (PIDTC), Rare Clinical Diseases Research Network, National Institutes of Health

Study record dates

Study Major Dates

• Study Start (Actual): February 2, 2014
• Primary Completion (Actual): May 1, 2019
• Study Completion (Actual): May 1, 2019

Study Registration Dates

• First Submitted: February 13, 2014
• First Submitted That Met QC Criteria: February 14, 2014
• First Posted (Estimate): February 17, 2014

Study Record Updates

• Last Update Posted (Actual): August 10, 2020
• Last Update Submitted That Met QC Criteria: August 6, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Keywords: Hematopoietic Stem Cell Transplantation; Wiskott-Aldrich Syndrome; Genetic Therapy
• Additional Relevant MeSH Terms: Pathologic Processes; Immunologic Deficiency Syndromes; Immune System Diseases; Disease; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Leukopenia; Leukocyte Disorders; Primary Immunodeficiency Diseases; Lymphopenia; Syndrome; Wiskott-Aldrich Syndrome
• Other Study ID Numbers: DAIT RDCRN PIDTC-6904