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

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

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.

Study Overview

• Status: Terminated
• Conditions: Thrombocytopenia; Stem Cell Transplant Complications
• Intervention / Treatment: Drug: N-acetyl-L-cysteine (NAC); Other: supportive therapy

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 Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100044
      • Peking University People's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Peking University People's Hospital
• Collaborators: Nanfang Hospital of Southern Medical University; The First Affiliated Hospital of Soochow University
• Investigators: Principal Investigator: Xiao-Jun Huang, MD, Peiking University

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2018
• Primary Completion (Actual): December 30, 2018
• Study Completion (Actual): December 30, 2018

Study Registration Dates

• First Submitted: January 2, 2018
• First Submitted That Met QC Criteria: January 4, 2018
• First Posted (Actual): January 5, 2018

Study Record Updates

• Last Update Posted (Actual): September 25, 2020
• Last Update Submitted That Met QC Criteria: September 23, 2020
• Last Verified: September 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Platelet Disorders; Thrombocytopenia; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Antiviral Agents; Protective Agents; Respiratory System Agents; Antioxidants; Antidotes; Free Radical Scavengers; Expectorants; Acetylcysteine; N-monoacetylcystine
• Other Study ID Numbers: 2017PHB220

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No