Thrombocytopenia, Stem Cell Transplant Complications
Conditions
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.
Interventions
NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant
OTHERsupportive therapy
prophylactic platelet transfusion was given when platelet count \<20000/ul
Sponsors
The First Affiliated Hospital of Soochow University
Nanfang Hospital, Southern Medical University
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
14 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| response rate | 30 days after the start of enrollement | rate of response after 30 days of treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| overall survival | the day of last follow-up | survival proportion at the last followup after transplantation |
| 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 |
| chronic graft versus host disease | the day of last follow-up | chronic graft versus host disease at the day of last follow-up |
Countries
China
Outcome results
None listed