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N-acetylcysteine as prophylactic therapy for transplant-associated thrombotic microangiopathy: a randomized, placebo-controlled trial
Pan, T., Qi, J., Tang, Y., Yao, Y., Chen, J., Wang, H., Yang, J., Xu, X., Shi, Q., Liu, Y., et al
Transplantation and cellular therapy. 2022
Abstract
BACKGROUND Transplant-associated thrombotic microangiopathy (TA-TMA) is a life-threatening complication for patients undergoing hematopoietic stem cell transplantation (HSCT). N-acetylcysteine (NAC) has recently been considered as a potential treatment for patients with thrombotic thrombocytopenic purpura. OBJECTIVES To assess the value of NAC for the prevention of TA-TMA, we conducted a prospective study at the First Affiliated Hospital of Soochow University. STUDY DESIGN This open-label, randomized placebo-controlled trial included 160 patients who were scheduled to receive allogeneic HSCT. Participants were randomly assigned 1:1 to either oral NAC (50 mg/kg daily from 9 days before HSCT to 30 days after HSCT) or placebo treatment. The primary outcome was the incidence of TA-TMA. Overall survival (OS) and event-free survival (EFS) were assessed between NAC and placebo cohorts. RESULTS The incidence of TA-TMA in the NAC cohort was 9.1% (95% confidence interval (CI), 0.02-0.162), compared with 23% (95% CI, 0.132-0.328) in placebo cohort, with a rate ratio of 0.34 (95% CI, 0.123-0.911; p=0.039). The median time to onset of TA-TMA was 60 (interquartile range [IQR] 42-129) and 36 (IQR 30.5-51) days in the NAC and control groups, respectively (p=0.063). The 2-year rates of OS in the NAC and placebo groups were 75.4% (95% CI, 2.865-7.353) and 63.0% (95% CI, 0.508-0.735), respectively, with a hazard ratio (HR) of 0.622 (95% CI, 0.334-1.155; p=0.132). The EFS rate was 25.8% in NAC patients and 8.1% in placebo patients, with an HR of 0.254 (95% CI, 0.094-0.692; p=0.024). The median time of EFS was 60 and 38 days in NAC and placebo cohorts. CONCLUSION Our findings suggest that NAC may be a potential treatment to reduce TA-TMA incidence.
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Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial
Tang, Y., Chen, J., Liu, Q., Chu, T., Pan, T., Liang, J., He, X. F., Chen, F., Yang, T., Ma, X., et al
Blood advances. 2021;5(5):1250-1258
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Abstract
Refractory prolonged isolated thrombocytopenia (RPIT) is an intractable complication after allogeneic hematopoietic cell transplantation (HCT), which often leads to poor prognosis. A clinical study was designed to validate the efficacy and safety of low-dose decitabine for RPIT after HCT and explore the related underlying mechanisms. Eligible patients were randomly allocated to receive 1 of 3 interventions: arm A, low-dose decitabine (15 mg/m2 daily IV for 3 consecutive days [days 1-3]) plus recombinant human thrombopoietin (300 U/kg daily); arm B, decitabine alone; or arm C, conventional treatment. The primary end point was the response rate of platelet recovery at day 28 after treatment. Secondary end points included megakaryocyte count 28 days after treatment and survival during additional follow-up of 24 weeks. Among the 91 evaluable patients, response rates were 66.7%, 73.3%, and 19.4% for the 3 arms, respectively (P < .001). One-year survival rates in arms A (64.4% ± 9.1%) and B (73.4% ± 8.8%) were similar (P = .662), and both were superior to that in arm C (41.0% ± 9.8%; P = .025). Megakaryocytes, endothelial cells (ECs), and cytokines relating to megakaryocyte migration and EC damage were improved in patients responding to decitabine. This study showed low-dose decitabine improved platelet recovery as well as overall survival in RPIT patients after transplantation. This trial was registered at www.clinicaltrials.gov as #NCT02487563.
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Oral cryotherapy for oral mucositis management in patients receiving allogeneic hematopoietic stem cell transplantation: a prospective randomized study
Lu, Y., Zhu, X., Ma, Q., Wang, J., Jiang, P., Teng, S., Zhou, L., Wu, D., Wang, H.
Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer. 2019
Abstract
PURPOSE To explore the best schedule of oral cryotherapy for the prevention of oral mucositis in recipients of myeloablative hematopoietic stem cell transplantation (HSCT). METHODS A prospective randomized study was conducted to recruit allogeneic HSCT recipients, who were then randomly allocated into four arms to accept the following: oral cryotherapy during the whole course (arm A) or second half of the course (arm B) of cytotoxic agents administration, regular oral cryotherapy twice a day (arm C), or conventional oral care without cryotherapy (arm D). Status of oral mucositis was daily assessed from the first day of conditioning to the 15th day post-HSCT. A myeloablative conditioning regimen was used which was composed of busulfan, cyclophosphamide, and cytarabine. RESULTS Totally 160 cases were consecutively enrolled in this study, and 145 cases were eligible for oral mucositis assessment. Both arm A and arm B were associated with a lower incidence and short duration of severe mucositis (≥ grade 3), although no statistical difference was found between these two groups (p = 0.463, p = 0.678). The highest incidence of severe mucositis was observed in arm C. Recovery of mucositis also had a significant diversity among the 4 arms (F = 4.133, p = 0.008). CONCLUSIONS Risk and outcome of severe oral mucositis could be ameliorated by oral cryotherapy during the administration of cytotoxic agents for allogeneic HSCT patients receiving non-radiation myeloablative conditioning regimen, and a half-course schedule could acquire a comparable efficacy compared with the whole-course schedule.
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Plerixafor and granulocyte-colony-stimulating factor for mobilization of hematopoietic stem cells for autologous transplantation in Chinese patients with non-Hodgkin's lymphoma: a randomized Phase 3 study
Zhu, J., Huang, H., Chen, H., Zhang, X., Li, Z., Wu, D., Zhou, D., Song, Y., Hu, Y., Liang, Y., et al
Transfusion. 2017
Abstract
BACKGROUND This Phase 3 randomized, double-blind study evaluated the efficacy and safety of plerixafor plus granulocyte-colony-stimulating factor for the mobilization of hematopoietic stem cells in Chinese patients with non-Hodgkin's lymphoma. STUDY DESIGN AND METHODS Adults (ages 18-75 years) with non-Hodgkin's lymphoma in first or second complete or partial remission, without previous hematopoietic stem cell mobilization or autologous transplant, were included. Patients received granulocyte-colony-stimulating factor 10 micro g/kg/day from Days 1 through 4 before they were randomized (1:1) to receive either plerixafor 0.24 mg/kg/day or placebo subcutaneously on Days 4 through 7 plus continued granulocyte-colony-stimulating factor on Days 5 through 8. Apheresis began on Day 5 and continued for no more than 4 days. The primary endpoint was collection of 5 x 106 CD34+ cells/kg or greater over no more than 4 days of apheresis. Other endpoints included the collection of 2 x 106 CD34+ cells/kg or greater and safety. RESULTS Overall, 101 patients were enrolled, and 50 were randomized to each group. More patients in the plerixafor group achieved 5 x 106 CD34+ cells/kg or greater (62 vs. 20%; p<0.0001) or 2 x 106 CD34+ cells/kg or greater (88 vs. 66%) and underwent transplantation (88 vs. 68%) compared with those in the placebo group. The most common plerixafor-related adverse events were nausea (7.8%) and diarrhea (3.9%). CONCLUSION Plerixafor plus granulocyte-colony-stimulating factor is superior to placebo plus granulocyte-colony-stimulating factor for the mobilization of CD34+ cells for autologous transplantation and is generally well tolerated in Chinese patients with non-Hodgkin's lymphoma.