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

Phase 2

Terminated

• Conditions: Thrombocytopenia; Stem Cell Transplant Complications
• Interventions: Drug: N-acetyl-L-cysteine (NAC); Other: supportive therapy

• Registration Number: NCT03391856
• Lead Sponsor: Peking University People's Hospital

Brief Summary

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

1. 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.)

2. 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.

3. 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.

Study Timeline

• Study First Submitted: 2018-01-02
• Study First Submitted QC: 2018-01-04
• Study First Posted: 2018-01-05
• Study Start: 2018-03-01
• Primary Completion: 2018-12-30
• Study Completion: 2018-12-30
• Status Verified: 2020-09
• Last Update Submitted: 2020-09-23
• Last Update Posted: 2020-09-25

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
intervention arm	N-acetyl-L-cysteine (NAC)	NAC 400mg p.o tid from day 60 to day 90 post transplant
controlled arm	supportive therapy	Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count \<20000/ul

Primary Outcome Measures

Name	Time	Method
response rate	30 days after the start of enrollement	rate of response after 30 days of treatment

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China