AZA Combined With NAC for PIT After HSCT
- Conditions
- Thrombocytopenia, IsolatedStem Cell Transplant Complications
- Interventions
- Drug: N Acetyl L Cysteine
- Registration Number
- NCT05126004
- Brief Summary
Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.
- Detailed Description
Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Furthermore, AZA had shown its potential in immune regulation. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 100
- Platelet count ≤ 30 × 10^9/L persistently at day 60 post-HSCT or later;
- Neutrophil and hemoglobin were well recovered;
- Full donor chimerism was achieved;
- Patients with malignancy relapse;
- Active infections;
- Grade Ⅲ-Ⅳ acute graft-versus-host disease or severe chronic graft-versus-host disease according to National Institute of Health criteria;
- Severe organ damage;
- Thrombosis requiring treatment;
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description AZA+NAC group Azacitidine AZA 50mg Subcutaneous daily d1-d5 + NAC 600mg oral bid d1-28， 28 days for one cycle AZA+NAC group N Acetyl L Cysteine AZA 50mg Subcutaneous daily d1-d5 + NAC 600mg oral bid d1-28， 28 days for one cycle
- Primary Outcome Measures
Name Time Method platelet reconstruction From date of randomization until the date of platelet reconstruction, assessed up to 100 days platelet count above 50\*10\^9/L independent of transfusion
- Secondary Outcome Measures
Name Time Method overall survival From date of randomization until the date of death from any cause, assessed up to 1 year the time from the date of day 1 post HSCT to the date of death due to any cause
overall response rate From date of randomization until the date of platelet count between 30*10^9/L and 50*10^9/L, assessed up to 100 days platelet count evaluating above 30\*10\^9/L but below 50\*10\^9/L independent of platelet transfusion
Trial Locations
- Locations (1)
The first affiliated hospital of Soochow University
🇨🇳Suzhou, Jiangsu, China