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This is a prospective single-arm clinical study to evaluate the role of NAC among patients with severe aplastic anemia (SAA) can promote hematopoietic recovery after haploidentical transplantation.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment of severe aplastic anemia (SAA). However, poor hematopoietic reconstitution including poor graft function (PGF) and prolonged isolated thrombocytopenia (PT), remains a life-threatening complication after allo-HSCT. Especially with the increasing use of haploidentical allo-HSCT (haplo-HSCT) in the past ten years, PGF and PT have become growing obstacles contributing to high morbidity and mortality after allo-HSCT. A previous clinical prospective cohort study showed that NAC could improve the function of bone marrow endothelial progenitor cells and promote hematopoietic recovery among leukemia patients after haploidentical transplantation. Therefore, we hypothesized that the prophylactic administration of NAC could facilitate the recovery of hematopoietic capacity by improving the bone marrow microenvironment of patients with SAA after haploidentical transplantation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NAC group | Experimental | Aplastic anemia patients receiving haploidentical transplantation will be enrolled, and NAC (400mg tid) will be administered orally from day 14 before conditioning until day +60 post-HSCT. The initial dose of NAC was 400mg orally three times daily (TID). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| N Acetyl L Cysteine | Drug | For subjects in the experimental intervention arm (NAC group), if the patients met the inclusion criteria on day 14 before conditioning, they received NAC from day 14 before conditioning until day +60 post-HSCT. The initial dose of NAC was 400mg orally three times daily (TID). In cases of grade 3 or worse AEs (not including hematologic recovery), dose modifications including dose reductions or interruptions were permitted at the physician's discretion. After the resolution of AEs, the dose was re-escalated from to 400mg TID. |
| Measure | Description | Time Frame |
|---|---|---|
| The incidence of prolonged thrombcytopenia (PT), which was assessed at +2M post-HSCT. | PT was defined as platelet count less than 20×109/L or a dependence on platelet transfusion with the engraftment of other cell lines(ANC>0.5×109/L and hemoglobin>70 g/L without transfusion support) beyond day +60 post-HSCT in the presence of complete donor chimerism (CDC). | Two months post-HSCT. |
| The incidence of poor graft function (PGF), which was assessed at +2M post-HSCT. | PGF was defined as the presence of 2 or 3 cytopenic counts (ANC≤0.5×109/L, platelet≤20×109/L, or hemoglobin≤70 g/L) for at least 3 consecutive days beyond day +28 post-HSCT with a transfusion requirement, related with hypoplastic-aplastic BM, in the presence of complete donor chimerism (CDC). | Two months post-HSCT. |
| Measure | Description | Time Frame |
|---|---|---|
| The cumulative incidences of transplantation related mortality (TRM). | Number of participants suffered TRM will be calculated. | Two months post-HSCT. |
| The cumulative incidences of Thrombotic Microangiopathy (TMA). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xiaojun Huang, M.D. | Contact | +861088326006 | xjhrm@medmail.com.cn | |
| Zhengli Xu, M.D. | Contact | +8601088326900 | xuzhengli0202@163.com |
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| ID | Term |
|---|---|
| D000111 | Acetylcysteine |
| ID | Term |
|---|---|
| D003545 | Cysteine |
| D000603 | Amino Acids, Sulfur |
| D013457 | Sulfur Compounds |
| D009930 | Organic Chemicals |
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|
Number of participants suffered TMA will be calculated.
| Two months post-HSCT. |
| The cumulative incidences of graft versus host disease (GvHD). | Number of participants suffered GvHD will be calculated. | Two months post-HSCT. |
| The cumulative incidences of overall survival (OS). | Number of participants survived for 1 year post diagnosed will be calculated. | One year post-HSCT. |
| Adverse reactions | Liver function, renal function, respiratory syndrom assessed by CTCAE v4.0 during oral administration of NAC. | Two months post-HSCT. |
| D001855 | Bone Marrow Diseases |
| D000596 |
| Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |