NCT03198195

Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

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

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 5 (actual)
• Sponsor: Capital Research Institute of Pediatrics · Other Government
• Sex: All
• Age: 5 Months – 10 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Wiskott-Aldrich Syndrome

Interventions

Type	Name	Description
PROCEDURE	cyclophosphamide	on days +3，+4,using cyclophosphamide 50mg/kg

Timeline

• Start date: 2015-03-10
• Primary completion: 2017-03-10
• Completion: 2020-07-10
• First posted: 2017-06-26
• Last updated: 2017-06-27