Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

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Brief Title

Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

Official Title

Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome

Brief Summary

      A protocol named as "CIP-2015" for patients with Wiskott-Aldrich Syndrome may reduce the rate
      of GvHD.

      The details of the protocal followed with:

        1. Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total.

        2. GvHD Prophylaxis:

      Rabbit antihuman thymocyte globulin 7.5 mg/kg post-transplant cyclophosphamide (CY) (50
      mg/kg.d on days +3 and +4) Cyclosporine or tacrolimus, mycophenolate mofetil, on days +5

Detailed Description

      Patients were enrolled in CIP-2015 Protocol at the Capital Institute of Pediatrics (Beijing).
      The conditioning regimen consisted of fludarabine (40 mg/m2) from days -6 to -3, and Busulfan
      was administered intravenously for 4 days, from days -5 to -2,using dose targeting based on
      therapeutic drug monitoring. Thymoglobulin (Sanofi, Cambridge, MA) 7.5 to 10 mg/kg (cumulative
      dose over 4 days) was administered over 4 days, from days -5 to -2. Bone marrow (BM) and PBSC
      were infused on day 0, followed by post-transplant CY (50 mg/kg/day, on days +3 and +4). To
      protect against hemorrhagic cystitis, MESNA (2-mercaptoethane sodium sulfonate) was
      administered at 150% of the CY dose. Post grafting immunosuppression with mycophenolate
      mofetil and tacrolimus commenced on day +5 and extended until days +28 and +84, respectively.
      Tacrolimus was tapered off by day +90 if there was no GVHD.

Study Type

Observational [Patient Registry]

Primary Outcome

Rate of aGvHD


Wiskott-Aldrich Syndrome




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

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

March 10, 2015

Completion Date

July 10, 2020

Primary Completion Date

March 10, 2017

Eligibility Criteria

        Inclusion Criteria:

        -Patients diagnosed with Wiskott-Aldrich Syndrome with indication of Hematopoietic stem
        cell transplantation

        Exclusion Criteria:

          -  without indication of Hematopoietic stem cell transplantation




5 Months - 10 Years

Accepts Healthy Volunteers



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Administrative Informations



Organization ID


Responsible Party

Principal Investigator

Study Sponsor

Capital Research Institute of Pediatrics

Study Sponsor

, , 

Verification Date

June 2017