ABSTRACT
Background: Hemophagocytic lymphohistiocytosis (HLH) is a rare complication following hematopoietic stem cell transplantation (HSCT). Currently, there is a lack of consensus recommendations for the treatment of post-transplant HLH. This case report emphasizes the successful utilization of ruxolitinib as a salvage therapy for HLH post-HSCT. The aim is to provide valuable insights into the optimal management of this rare and complex complication. Case Description: We present a case study of an 11-year-old male patient diagnosed with severe aplastic anemia who received a haploidentical HSCT. On the 86th day post-transplantation, the patient developed recurrent fever, hepatomegaly, hypertriglyceridemia, severe pancytopenia, and elevated levels of inflammatory factors and ferritin. Hemophagocytosis was observed in the bone marrow, and subsequent DNA next-generation sequencing identified adenovirus type C infection, leading to a diagnosis of adenovirus-associated HLH. After unsuccessful treatment attempts with cidofovir, dexamethasone, immunoglobulin, plasmapheresis, and etoposide, ruxolitinib was administered. Remarkably, the patient's clinical symptoms rapidly improved, and his test results gradually normalized with ruxolitinib therapy. The adenovirus viral load became undetectable by the 180th day. With continuous remission, ruxolitinib was discontinued on the 137th day post-transplantation, and a 15-month follow-up examination showed no relapse. Conclusions: We present a case of adenovirus-related secondary HLH (sHLH) post-HSCT, which was effectively treated with ruxolitinib. Our case highlights the potential of ruxolitinib as a therapeutic option for patients with viral infections and sHLH. Nonetheless, the safety and efficacy of this innovative treatment should be evaluated in forthcoming large-scale clinical trials.
ABSTRACT
Background: Undifferentiated embryonal sarcoma of the liver (UESL) is a rare and highly aggressive malignancy predominantly affecting children and adolescents. Managing UESL is particularly intricate due to its aggressive nature and the limited array of treatment options available. This study is dedicated to elucidating the efficacy of a multimodal therapeutic strategy in the successful management of UESL. Case Description: Four pediatric patients (two males, two females; aged 6-11 years) diagnosed with UESL were treated at the Children's Hospital of Nanjing Medical University between November 2019 and June 2023. Surgical resection with lymph node dissection achieved complete primary tumor eradication. Adjuvant chemotherapy tailored to each patient's needs was followed by localized radiation therapy. After 9-42 months of follow-up, one patient who did not undergo immediate radiotherapy experienced a relapse. Following a second operation coupled with radiotherapy, the patient achieved complete remission, and mirroring the status of the other three patients who are now presently in remission. The overall cohort exhibited commendable tolerance to the treatment regimen, with manageable chemotherapy-related toxicities. Conclusions: This case series suggests that implementing a standardized protocol of resection, followed by adjuvant chemotherapy and radiation, can lead to favorable outcomes in pediatric patients diagnosed with UESL. Nevertheless, the need for comprehensive large-scale studies is imperative to substantiate the effectiveness of this approach.