Background: Haploidentical stem cell transplantation (haplo-SCT) is an option for patients without human leukocyte antigen-matched related or unrelated donor. Post-transplantation cyclophosphamide (PTCy) is an effective method of graft versus host disease (GVHD) prophylaxis and permits the use of T-cell replete grafts in settings were ex vivo manipulation is not feasible. Methods: A retrospective study among patients younger than 18 years, with a history of hematologic malignancies who underwent haplo-SCT between 2012 and 2016. All patients received a preparative regimen of fludarabine, busulfan, and 400 cGy total body irradiation or melphalan. Post-transplant GvHD prophylaxis consisted either of PTCy (50 mg/kg on Days + 3 and + 4) and cyclosporine (CSA) plus mycophenolate (MMF) (15 mg/kg/dose, thrice daily, per os), or minidose methotrexate (MTX; 5 mg/m2 dose) on Days + 5, +7, +10, and + 15. Results: A +total of 52 children were included, whose median age was 9 years (interquartile range, 4.9–14; range, 1.2–17 years), and 63% were males. The most common complications were cytomegalovirus reactivation (57%) and hemorrhagic cystitis (36%). The acute GVHD prophylaxis was PTCy, CSA, and mini-dose MTX in 42 (81%) patients, and 10 (19%) patients received PTCy, CSA, and MMF. The cumulative incidence of acute GvHD II–IV, acute GvHD III–IV, and chronic GvHD were 42%, 8.5%, and 19%, respectively. Grades I–IV acute GvHD occurred in 100% of the patients who received prophylaxis with CSA and MMF, and 62% who received CSA and mini-dose MTX (p = .055). The transplant-related mortality at 100 days was 18%. The 5-year overall and event-free survival were 59% and 57%, respectively. Conclusions: Haplo-SCT with PT/Cy can be an available, safe, and feasible option for children with hematologic malignancies; meanwhile, the use of mini-dose of MTX was associated with lower rates of acute GVHD. However, our results require further support from prospective randomized studies to improve the efficacy of this prophylactic strategy.
Gastrointestinal (GI) GVHD after allo-SCT is diagnosed on the basis of symptoms and findings in endoscopic mucosal biopsy specimens. However, GI symptoms often persist despite treatment and whether a second endoscopy may be helpful in determining the most suitable therapy is not established. We identified 31 patients with persistent diarrhea who underwent more than one endoscopic study. All cases underwent serial microbiological stool analysis and CMV-detecting assays on serum and biopsies. Of the 31 initial GI biopsies, 20 (64.5%) were classified as GVHD, two (6.5%) as GVHD with CMV, four (13%) as non-CMV infection, and five (16%) as normal or unspecific. The second GI biopsies were diagnostic of GVHD in nine cases (29%), GVHD simultaneously with CMV infection in four (13%), regenerative changes post-GVHD in five (16%), CMV infection in four (13%), and normal or unspecific in nine (29%). In 22 of the 31 patients (71%), the histological findings of the second/third endoscopic biopsies differed from the findings of the first endoscopy and led to a therapy change in 77%. In conclusion, serial GI endoscopies are of reliable diagnostic value and can impact on therapeutic decision-making for patients with persistent diarrhea after allo-SCT.
Introducción: En pacientes con leucemia mieloide aguda (LMA) el trasplante de progenitores hematopoyético (TPH) es el único tratamientoz curativo. El objetivo de este estudio es presentar la experiencia y resultados del trasplante haploidéntico en pacientes adultos con LMA en la Fundación Valle del Lili, Cali – Colombia.Materiales y métodos: Estudio de cohorte retrospectivo de pacientes que recibieron trasplante haploidéntico entre 2013 y 2017, con acondicionamiento mieloablativo y ciclofosfamida postrasplante, en Fundación Valle del Lili, Cali (Colombia).Resultados: Se realizaron 47 trasplantes en pacientes con leucemia mieloide aguda en la fecha de estudio, se incluyeron en el análisis 21 pacientes con donante haploidéntico, a 3 años tanto la supervivencia global y libre de eventos fue del 38%. La incidencia acumulada de mortalidad relacionada al trasplante fue del 26% a 100 días y del 38,3%, a 38 meses de seguimiento. La incidencia acumulada de recaída a 38 meses fue del 19%. Con respecto a la enfermedad injerto versus huésped (EICH) se encontró que la incidencia acumulada de EICH aguda grado II-IV, grado III-IV y EICH crónico fue del 19%, 5% y 19% respectivamente.Conclusión: Los resultados de este estudio sugieren que el trasplante haploidéntico es una alternativa factible como tratamiento para pacientes con diagnóstico de LMA en nuestro medio.
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