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

Peking University

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Thrombocytopenia; Stem Cell Transplant Complications

Treatments: Other: supportive therapy; Drug: N-acetyl-L-cysteine (NAC)

Study type: Interventional

Funder types: Other

Identifiers

NCT03391856

2017PHB220

Details and patient eligibility

About

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.

Full 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.

Enrollment: 15 patients

Sex: All

Ages: 14+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

15 participants in 2 patient groups

intervention arm

Experimental group

Description:

NAC 400mg p.o tid from day 60 to day 90 post transplant

Treatment:

Drug: N-acetyl-L-cysteine (NAC)

controlled arm

Other group

Description:

Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count \<20000/ul

Treatment:

Other: supportive therapy