Prolonged Isolated Thrombocytopenia After Allo-SCT : N-acetyl-L-cysteine (NAC) Versus Supportive Therapy

Study Overview
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Brief Summary
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Isolated prolonged thrombocytopenia (PT) is a common complication after allogeneic stem cell transplantation with significant poor prognosis. No standard treatment is available. The current study assigned PT randomly to 2 arms: intervention arm with N-acetyl-L-cysteine (NAC) and control arm with supportive therapy.This is a prospective randomized controlled study.

Detailed Description
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patients diagnosed with PT at day 60 post transplant will be randomized assigned to intervention arm (NAC) or controlled arm (supportive therapy: prophylactic platelet transfusion was given when platelet count <20000/ul.) Response will be evaluated at day 90. Response was defined as platelet recovery to >= 20000/ul for 7 consecutive days without transfusion support during the enrollment period. All the other patients not achieved above criteria was defined as no response. For those without response in both arms, patients will received NAC plus recombinant human thrombopoietin (rhTPO) for an additional 30 days. rhTPO was given at 300IU/kg/d for 28 consecutive days or platelet > 50000/ul independent of platelet transfusion.

Official Title
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A Prospective Randomized Controlled Study for Intervention of Prolonged Isolated Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplantation: N-acetyl-L-cysteine (NAC) Versus Supportive Therapy

Conditions
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Thrombocytopenia, Stem Cell Transplant Complications

Intervention / Treatment
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* Drug: N-acetyl-L-cysteine (NAC)
* Other: supportive therapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: age>= 14 years diagnosed as PT (defined as platelet count in peripheral blood never achieve 20000/miro-liter for 7 consecutive days without transfusion at day 60 post transplantation) serum creatine level < ULN (upper limit of normal), serum ALT/AST /TBil<=2 ULN without active CMV/EBV/ADV reactivation, active GVHD, without relapse or minimal residual disease at enrollment Exclusion Criteria: history of asthma allergy to NAC refuse to participate

Ages Eligible for Study
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Minimum Age: 14 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: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: intervention arm<br>NAC 400mg p.o tid from day 60 to day 90 post transplant | Drug: N-acetyl-L-cysteine (NAC)<br>* NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant<br>|
| Other: controlled arm<br>Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count <20000/ul | Other: supportive therapy<br>* prophylactic platelet transfusion was given when platelet count <20000/ul<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| response rate | rate of response after 30 days of treatment | 30 days after the start of enrollement | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| overall survival | survival proportion at the last followup after transplantation | the day of last follow-up | 
| non-relapse mortality | non-relapse mortality at the last followup after transplantation | the day of last follow-up | 
| grade 2-4 acute graft versus host disease | grade 2-4 aGVHD at day 100 after transplant | day 100 after transplant | 
| chronic graft versus host disease | chronic graft versus host disease at the day of last follow-up | the day of last follow-up |