Hematopoietic stem cell transplantation for children with β-thalassemia major: multicenter experience in China

Backgroundβ-Thalassemia major (β-TM) has become a public health problem in China's Mainland. Hematopoietic stem cell transplantation (HSCT) has remained the only cure forβ-TM in China's Mainland since 1998. Methods This multicenter retrospective study provides a comprehensive review of the outcomes of 50 pediatric patients withβ-TM who received HSCT between 1998 and 2009 at five centers in China's Mainland. Both related (n = 35) and unrelated donors (n = 15) with complete human leukocyte antigen matches were included. The stem cell sources included bone mar-row (BM), peripheral blood stem cells, umbilical cord blood (UCB) and a combination of BM and UCB or a combination of BM and peripheral blood stem cells from a single sibling donor. Results The probabilities of 5-year overall survival (OS) and thalassemia-free survival (TFS) after the first HSCT were 83.1 and 67.3%, respectively. Graft failure (GF) occurred in 17 patients. Univariate analyses showed that umbilical cord blood transplantation (UCBT) was one of the potential risk factors for decreased OS (P = 0.051), and that UCBT (P = 0.002) was potentially related to TFS. GF incidence was distinct between the UCBT and non-UCBT groups (P = 0.004). Four cases of UCB-BM combined transplantation led to decreased risks of mortality and recurrence. In the UCBT group, related donor transplantation produced more favorable results than unrelated donor transplantation in OS (P = 0.009) but not in TFS (P = 0.217). Conclusions GF was the primary cause of UCBT failure. Though UCBT from related donors was not favorable, the combined transplantation of UCB and BM could improve the prognosis of UCBT.

Haploidentical hematopoietic stem cell transplantation for pediatric patients with chronic active Epstein-Barr virus infection:a retrospective analysis of a single center

Background This study aimed to evaluate the feasibility and clinical effect of haploidentical hematopoietic stem cell transplantation(haplo-HSCT)for the treatment of pediatric patients with chronic active Epstein-Barr virus infection(CAEBV).Methods Children with CAEBV who did not have matched donors and underwent haplo-HSCT in Beijing Children's Hospital,Capital Medical University,from October 2016 to June 2020 were analyzed retrospectively.Data relating to the clinical manifestations,engraftment,and prognosis of the children were extracted from medical records.Results Twenty-five patients,including 16 males and 9 females,with an onset age of 5.0±2.6 years and a transplantation age of 6.9±2.9 years,were enrolled irnhis study.The mean time from diagnosis to transplantation was 3.8(2.0-40.2)months.The mean observation time was 19.0±12.0 months.Three patients received the reduced intensity conditioning regimen,and the remaining patients all received the modified myeloablative conditioning regimen.By the end of the follow-up,23 patients were characterized by disease-free survival(DFS),22 were characterized by event-free survival(EFS).and two died.One of the patients died of thrombotic microangiopathy(TMA),and another died of graft versus host disease(GVHD);this patient discontinued the treatment for economic reasons.The 3-year overall survival(OS)rate was estimated to be 92.0%±5.4%,and the 3-year EFS rate was estimated to be 87.4%±6.8%.All active patients survived after HSCT event-free.Acute GVHD degrees 1-3 were observed in ten patients(40.0%),and degree IV was observed in six(24.0%),who were all cured except for one patient.Chronic GVHD was observed in nine(36.0%),and most of these cases were mild.The incidence of TMA and veno-occlusive disease(VOD)was 28.0%and 4.0%.Conclusions Haploidentical hematopoietic stem cell transplantation is safe and effective in the treatment of pediatric CAEBV and can be used as an alternative therapy without matched donors or emergency transplantation.Patients with active disease before HSCT also benefited from haplo-HSCT.Haplo-HSCT requires careful monitoring for complications,such as GVHD and TMA.Early detection of TMA and timely treatment can reduce mortality and can improve the survival rate.

Infections During Induction Therapy of Protocol CCLG-2008 in Childhood Acute Lymphoblastic Leukemia： A Single-center Experience with 256 Cases in China

Background：Infections remain a major cause of therapy-associated morbidity and mortality in children with acute lymphoblastic leukemia （ALL）.Methods：We retrospectively analyzed the medical charts of 256 children treated for ALL under the CCLG-2008 protocol in Beijing Children＇s Hospital.Results：There were 65 infectious complications in 50 patients during vincristine,daunorubicin,L-asparaginase and dexamethasone induction therapy,including microbiologically documented infections （n =12; 18.5％）,clinically documented infections （n =23; 35.3％） and fever of unknown origin （n =30; 46.2％）.Neutropenia was present in 83.1％ of the infectious episodes.In all,most infections occurred around the 15t1h day of induction treatment （n =28）,and no patients died of infection-associated complications.Conclusions：The infections in this study was independent of treatment response,minimal residual diseases at the end of induction therapy,gender,immunophenotype,infection at first visit,risk stratification at diagnosis,unfavorable karyotypes at diagnosis and morphologic type.The infection rate of CCLG-2008 induction therapy is low,and the outcome of patients is favorable.

Clinical characteristics of herpes zoster in a pediatric hospital in China from 2007 to 2020

Herpes zoster(HZ)also known as shingles is caused by thereactivationof latentvaricella zostervirus(VZV)from the dorsal root ganglia following primary infection[1,2]and is associated with severe disease in immunocompromised pediatric patients[3-6].Immunosuppression has been demonstrated to be related to a higher incidence of HZ[4].The prevalence and clinical features of HZ in the hospitalization of patients who have a history of other medical conditions has been studied[7].