Clinical trial NCT03391856: Prolonged Isolated Thrombocytopenia After Allo-SCT : N-acetyl-L-cysteine (NAC) Versus Supportive Therapy
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Thrombocytopenia
Stem Cell Transplant Complications
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
Collaborators: name: The First Affiliated Hospital of Soochow University
name: Nanfang Hospital, Southern Medical University
First posted: 2018-01-05
Enrollment: 15
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: response rate
timeFrame: 30 days after the start of enrollement
Secondary outcomes:
measure: overall survival
timeFrame: the day of last follow-up
measure: non-relapse mortality
timeFrame: the day of last follow-up
measure: grade 2-4 acute graft versus host disease
timeFrame: day 100 after transplant
measure: chronic graft versus host disease
timeFrame: the day of last follow-up
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peiking University
name: Xiao-Jun Huang, MD
role: PRINCIPAL_INVESTIGATOR
References: