Background Subcutaneous panniculitis T-cell lymphoma (SPTCL) is a rare, cytotoxic T-cell lymphoma with which some patients have accompanying hemophagocytic syndrome (HPS). There is currently no standard treatment regimen. In the past, the most commonly used treatment was multidrug chemotherapy. In contrast, numerous case reports or small series suggest that immunosuppressive drugs could also be effective for some patients. Since this NHL subtype is extremely rare in children and adolescents, to improve the understanding of this disease and standardize its rational treatment, we retrospectively summarized the treatment regimens of 18 pathologically diagnosed children with SPTCL to compare the clinical efficacy of multidrug chemotherapy and immunomodulatory therapy. Results The median age of onset was 11.1 years. Painless subcutaneous nodules or skin patchy lesions were found in all patients, most commonly involving the lower extremities and/or trunk. Before January 1, 2019, the treatment was mainly chemotherapy, and 10 patients were initially treated with chemotherapy, among whom was one patient who progressed during initial treatment, was voluntarily discharged and was subsequently lost to follow-up, one patient who died of disease progression, and the remaining 8 patients who all achieved sustained remission, with a complete remission (CR) rate of 80% (8/10). Corticosteroids combined with cyclosporine A or ruxolitinib were the most common initial immunosuppressive agents at our center after January 1, 2019 and had a CR rate of 71.4% (5/7). In addition, 1 patient achieved partial remission (PR) during follow-up, and one had autologous hematopoietic stem cell transplantation (AHSCT) after 4 months of drug withdrawal. There were 7 patients (38.9%, one case in chemotherapy group and six cases in immunotherapy group) with HPS and 4/5 screened patients (80%) with positive HAVCR2 gene mutations. The median follow-up was 17 months. Conclusion The prognosis of SPTCL is relatively good. Previous multi-drug and long-term chemotherapy treatment has clear efficacy, and recent immunomodulatory therapy as pre-chemotherapy therapy can also benefit patients.
Introduction Despite rasburicase's proven efficiency in Caucasians, Japanese, and Koreans, studies evaluating the safety and effectiveness of rasburicase in Chinese pediatric patients with non-Hodgkin’s lymphoma (NHL) and acute leukemia (AL) in particular are lacking. Objective The aim was to evaluate the safety and effectiveness of rasburicase in Chinese pediatric patients with NHL and AL. Methods In this phase IV, open-label, non-randomized, single-arm, multi-center, interventional study (NCT04349306), children newly diagnosed with NHL or AL who received 0.20 mg/kg/day of rasburicase were included. The primary objective was to assess the safety of rasburicase by the incidence of adverse events (AEs). The secondary objective was to determine the effectiveness of rasburicase in the control of hyperuricemia. Results Out of 50 patients, 25 reported a total of 76 treatment-emergent adverse events (TEAEs), including eight TEAEs of grade ≥ 3 in 12 patients. A drug-related serious AE was reported in one patient, and there was no incidence of death. The response rate in the intent-to-treat population was 100.0% (95% confidence interval 82.4–100.0) in patients ( n = 19) with baseline uric acid level of > 8.0 mg/dL. Similarly, the response rate was 86.2% ( n = 25) among 29 patients (60.4%) with baseline uric acid levels of ≤ 8.0 mg/dL. The maximum mean percentage decrease of plasma uric acid level in the overall patients was 96.9%. Conclusion Rasburicase was well tolerated and effective in controlling hyperuricemia in Chinese pediatric patients with NHL and AL. Supplementary Information The online version contains supplementary material available at 10.1007/s40268-023-00420-y.
Purpose We intended to investigate the clinical features of pediatric patients with chronic active Epstein-Barr virus infection (CAEBV) and effectiveness of the L-DEP regimen before HSCT (hematopoietic stem cell transplantation).Methods A retrospective analysis was performed on 35 patients with CAEBV at Beijing Children’s Hospital from January 2016 to January 2020. The efficacy and adverse events of the L-DEP regimen were evaluated.Results The median age of 35 patients was 7.0 years (range 2.5-17.5 years). 28 patients achieved clinical response (80.0%, 22 in clinical CR, 6 in clinical PR) after L-DEP. In terms of virological response, 7 patients (20%) were assessed as virological CR and 23 patients (65.7%) were virological PR. Finally, 29 patients underwent allo-HSCT. Median survival time was 18 months (2-50 months). The 3-year overall survival rates in patients treated with chemotherapy only (n=6), chemotherapy followed by HSCT (n=25) were 33.3% and 75.4%, respectively. After L-DEP 1st treatment, the amount of EBV-DNA loads in blood and plasma were significantly reduced than that before chemotherapy (median: 4.29×105 vs. 1.84×106, Mann-Whitney U:P=0.0004; 5.00×102 vs. 3.17×103, Mann-Whitney U:P=0.003). And, compared with liver and spleen size before chemotherapy, the size of liver and spleen shrank significantly after L-DEP 2nd (median 3.8cm vs. 1.9cm, P=0.003; 3.8cm vs. 0cm, P<0.008). In addition, after L-DEP treatment, there was no difference in clinical or virological response rate whether HLH was accompanied or not (clinical response: 77.3% vs. 84.6%:P=0.689; virological response: 90.9% vs. 76.9%, P=0.337). Conclusion L-DEP regimen is an effective therapy in treating CAEBV for bridging to allo-HSCT.
ObjectiveTo evaluate the clinical efficacy of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for treatment of malignant infantile osteopetrosis (MIOP) and intermediate osteopetrosis.MethodsChildren with MIOP and IOP who underwent haplo-HSCT in Beijing Children’s Hospital, Capital Medical University, from January 2010 to May 2018 were retrospectively analyzed. Data relating to the clinical manifestations, engraftment, and prognosis of the children were extracted from medical records. ResultsTwenty-seven patients, including 18 males and 9 females, with an onset age of 12 (0.04-72) months, were enrolled in this study. The median time from diagnosis to transplantation was 4 (1-23) months. All patients received haplo-HSCT with myeloablative conditioning regimen (including fludarabine, busulfan, and cyclophosphamide). The graft versus host disease (GVHD) prophylaxis was based on anti-human T lymphocyte porcine immunoglobulin /anti-human thymus globulin, methotrexate, and mycophenolate mofetil. The median observation time was 55.2 (0.3-126.2) months. By the end of follow-up, twenty patients survived and seven patients died. The five years overall survival rate was 73.9%. Acute GVHD degree I-II was observed in 20 patients, degree III in 1 patient and without degree IV. Chronic GVHD was observed in 11 patients (40.7%). It was controlled by anti-GVHD therapy.ConclusionHaplo-HSCT was effective for MIOP and IOP, with high survival rate and significantly improved of clinical symptoms. For the patients with vision impairment before HSCT, the improvement was slow after transplantation. The incidence of GVHD was high but mild, and could be effectively controlled after appropriate treatment. These data provided that haplo-HSCT was feasible in the treatment of MIOP and IOP.
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