2023, Vol. 27 ›› Issue (1): 42-48

Complications and death causes of peripheral blood stem cell transplantation in the treatment of thalassemia major

Huang Chuwen1, Jiang Hua1, Li Minqing2   

Abstract: BACKGROUND: Allogeneic hematopoietic stem cell transplantation is the only effective method to cure thalassemia major. However, the risk factors influencing the prognosis of transplantation remain unclear.
OBJECTIVE: To analyze the distribution of common complications and cause of death after allogeneic hematopoietic stem cell transplantation in children with thalassemia major, and to explore the prognostic factors so as to provide references for improving the survival rate of patients.  
METHODS: This retrospective cohort study included 257 patients with β-thalassemia major who underwent allogeneic hematopoietic stem cell transplantation at Department of Hematology and Oncology, Guangzhou Women and Children Medical Center between September 2013 and September 2019. There were 172 males and 85 females, with a median age of 6 years at the time of transplantation. The basic clinical data before transplantation and complications after transplantation were compared between the surviving group and dead group using single factor. The overall survival rate was analyzed by the Kaplan-Meier method, and the overall survival rate was compared by the Log-rank test. Multivariate Cox regression was used to analyze factors affecting survival.
RESULTS AND CONCLUSION: The median follow-up time was 29 months, and no cases were lost to follow-up. (1) Univariate analysis results showed that there were significant differences in the risk classification between surviving patients and dead patients (P=0.033). Patients with higher risk class had an increased risk of death after transplantation. Multivariate analysis showed that risk classification and severe pneumonia were independent risk factors for overall survival after thalassemia major hematopoietic stem cell transplantation (P < 0.05). (2) Among 20 dead patients, there were 13 patients with severe pneumonia and respiratory failure, 2 patients with grade IV intestinal graft-versus-host disease, 1 patient with intracranial hemorrhage due to thrombocytopenia, 1 patient with thrombocytopenia with acute pulmonary hemorrhage, 1 patient with sepsis with shock, and 1 patient with myasthenia gravis. (3) Totally 17 cases died within 1 year after transplantation, while the rest 3 patients died of severe pneumonia more than 1 year after transplantation. (4) The incidence of bronchiolitis obliterans was significantly higher in patients who died after transplantation than in those who survived (P < 0.000 1). (5) It is concluded that the important factors that affect survival rate in thalassemia major patients after hematopoietic stem cell transplantation are infection and severe graft-versus-host disease. The risk of death increased in patients with bronchiolitis obliterans after transplantation.  
Key words: thalassemia major, hematopoietic stem cell transplantation, infection, graft-versus-host disease, survival rate