Improved Post-Transplant Cyclophosphamide Regimens for Pediatric Patients With Refractory AML

Study Overview
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Brief Summary
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No more datas about post-transplant cyclophosphamide (PT/Cy) used in pediatric refractory acute myeloid leukemia (R-AML）patients. Investigators reasoned that this group of patients if they have been treated with ablative conditioning regimens for HSCT combined with PT/Cy, under the rapid of immune reconstitution will have better outcomes.

Detailed Description
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Haploidentical stem cell transplantation (HSCT) is a potentially curative therapy for patients with high risk or refractory acute myeloid leukemia (R-AML). Graft-versus-host disease （GVHD）is a major barrier to achieve success for patients with HSCT. High dose cyclophosphamide given after HLA-matched related and unrelated allogeneic stem cell transplantation for patients with hematologic malignancies is effective single agent (GVHD) prophylaxis in adults and in pediatric benign patients. Data describing outcomes for pediatric maligant patients has not been reported. Investigators have recruited 26 pediatric patients (3 years < age <18 years) between March 2015 to July 2018 with primary induction R-AML treated in investigators' institution for R-AML with modified myeloablative regimen, post-transplant cyclophosphamide (PTCy) has been used as GVHD prophylaxis. Conditioning regimen of the group of patient consisted Idrabine(IDA,10mg/m2/day) which was administered intravenously for 2 days, from days -14 to -13, total body irritation (TBI, 9 Gy) which was divided into 3 fractions and 3 days from -12 to -10, thymoglobulin (2.5mg/kg/day) was administered for 3 days, from -9 to -7, etoposide (VP-16, 300mg/m2) which was infused intravenously on day -6, cladribine (10mg/m2/day) which was administered for 3 days from -5 to -2 .

Official Title
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Improved Post-Transplant Cyclophosphamide Regimens for Unmanipulated Haploidentical Transplant in Pediatric Patients With Refractory Acute Myeloid Leukemia

Conditions
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Hematopoietic System--Cancer

Intervention / Treatment
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* Drug: Cyclophosphamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: -refractory AML Exclusion Criteria: - complete remission AML

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 18 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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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cyclophophamide<br>Cyclophosphamide 50mg/kg/day on day+3，+4 after HSCT. Intervention: drugs:Cyclophosphamide. | Drug: Cyclophosphamide<br>* cyclophophamide<br>* Other names: Cy;|
| Experimental: Placebo<br>5% GLS（Placebo) 50ml/day on day+3，+4 after HSCT. Intervention: drugs: Placebo other name: placebo (for Cyclophosphamide) 5%Glugose in water 50ml or normal saline | Drug: Cyclophosphamide<br>* cyclophophamide<br>* Other names: Cy;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Disease status | Disease status can be measured by test the of (minimal residual disease) MRD via flow cytometry one time each month until one year after HSCT. MRD<0.0001 （complete remission), if not, relapse. | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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HSCT, AML， R-AML