Clinical analysis of the effect of fludarabine combined with post-transplantation cyclophosphamide in haploid hematopoietic stem cell transplantation / 中国临床药理学与治疗学

AIM: To investigate the therapeutic method of fludarabine combined with subsequent cyclophosphamide in the prevention of GVHD in haploid hematopoietic stem cell transplantation. METHODS: A total of 52 patients receiving PTCY (50 mg/kg, on days 3 and 4) were matched with 29 patients receiving ATG. RESULTS: The median follow-up time was 359 days. Complete donor chimerism was achieved in all patients by STR DNA detection on days +30, +60. The median time to neutrophils ≥0.5×10

Desenlace de los pacientes pediátricos con falla medular tratados en un centro de alta complejidad / Outcomes in pediatrics patients diagnosed with bone marrow failure disorders treated in a tertiary care center

INTRODUCCIÓN: Los síndromes de falla medular (SFM) son trastornos infrecuentes, con una incidencia anual de 2-4 casos por millón. Las opciones de tratamiento incluyen terapia de inmunosupresión (TIS) y restaura ción de la hematopoyesis con trasplante de progenitores hematopoyéticas (TPH). OBJETIVO: Analizar los desenlaces de pacientes pediátricos diagnosticados con SFM tratados en una institución de alta complejidad. PACIENTES Y MÉTODO: Estudio retrospectivo de pacientes pediátricos con diagnóstico de SFM que consultaron a la Fundación Valle del Lili, Cali. Se realizo análisis estadístico descriptivo según SFM adquirida (SFMA) y SFM congénita (SFMC). Los desenlaces incluyeron: tratamiento, complicaciones, supervivencia global (SG) en los trasplantados, calculada con el método Kaplan Meier. RESULTADOS: Se incluyeron 24 pacientes con SFM, edad 6,5 ± 4 años, 50% mujeres. El 58% fue ron SFMC, 9 con anemia de Fanconi, 2 disqueratosis congénita, 2 trombocitopenia amegacariocítica congénita, uno anemia Diamond-Blackfan. Doce pacientes con TPH tuvieron SG a 5 años de 83%. SFMA correspondió al 42%, 6 recibieron TIS-TPH, 3 TIS y 1 TPH, la SG del grupo con TIS-TPH fue 86%. Seis pacientes fallecieron, 4/6 relacionadas con infección. CONCLUSIONES: En esta serie fue mayor el número de casos con SFMC. La SG de los pacientes llevados a TPH es comparable con la reportada en estudios recientes. La causa de muerte predominante fue infecciosa que también se ha reportado previamente. El tratamiento instaurado en los pacientes de esta serie mostró resultados favorables en un centro de alta complejidad en un país latinoamericano.

INTRODUCTION: Bone marrow failure (BMF) syndromes are rare disorders with an annual incidence of 2-4 cases per million. Treatment options include immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT). OBJECTIVE: To analyze the outcomes of pediatric patients diagnosed with BMF treated in a tertiary care center. PATIENTS AND METHODP: Retrospective study of pediatric patients diagnosed with BMF who consulted at Fundación Valle de Lili, Cali. Descriptive statistical analysis was performed according to Acquired BMF (ABMF) and Inherited BMF (IBMF). The outcomes include treatment, complications, overall survival (OS) in transplant patients, calculated using the Kaplan Meier method. RESULTS: We included 24 patients with BMF, average age 6.5 ± 4 years, and 50% were women. 58% presented IBMF, 9 with Fanconi anemia (FA), 2 dyskeratosis congenita, 2 congenital amegakaryocytic thrombocytopenia, and 1 presented Diamond-Blackfan anemia. 12 patients treated with HSCT had a 5-year OS of 83%. ABMF represented 42%. 6 patients received IST-HSCT, 3 received IST, and 1 received HSCT. The OS of the IST-HSCT group was 86%. Six patients died, four of them related to infection. CONCLUSIONS: In this series, there was a higher number of cases with IBMF. The OS of patients treated with HSCT is similar to that reported in recent studies. The most frequent cause of death was of infectious origin which has also been previously reported. The treatment esta blished in the patients showed favorable results in a Latin American tertiary care center.

Optimal Dose of Thymoglobulin for Induction Therapy in High Risk Kidney Transplant Recipients / 대한이식학회지

BACKGROUND: Thymoglobulin has been used for induction therapy to prevent acute rejection and delayed graft function (DGF) in kidney transplant patients. However, the usual dose of thymoglobulin is considered to be related with frequent infection. We compared the efficacy and safety of low-dose thymoglobulin to high-dose treatment in high risk recipients with kidney transplantation. METHODS: Twenty-one kidney transplant recipients underwent induction treatment with thymoglobulin and were divided into two groups: patients treated with low-dose (<6.0 mg/kg) and high-dose thymoglobulin (≥6.0 mg/kg). All patients showed one or more risk factors for acute rejection or DGF. The risk factors were re-transplantation, recipient or donor age over 60 years, human leukocyte antigen full mismatch, and panel-reactive antibody more than 50%. We compared incidence of acute rejection, infection, hematologic complications, and graft survival between two groups. RESULTS: The demographic characteristics of the two groups were comparable. Mean follow-up duration was 11.9±4.3 months, and cumulative thymoglobulin dosage was 6.3±1.6 mg/kg. The incidence rates of acute antibody-mediated rejection (AMR), DGF and infectious events as cytomegalovirus disease, or urinary tract infection were not significantly different between the two groups. Neutropenia occurred more frequently in the high-dose thymoglobulin group, but there was no statistically significant difference. The rate of graft loss were similar between the two groups. CONCLUSIONS: There were no differences in graft survival, infectious disease, and hematologic problems between the two groups. We suggest to lower the dose of thymoglobulin to less than 6 mg/kg for prevent acute AMR and DGF in high risk patients.