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1.
Pediatr Hematol Oncol ; : 1-15, 2024 Sep 23.
Artigo em Inglês | MEDLINE | ID: mdl-39310983

RESUMO

Anti-thymocyte globulin (ATG) forms an essential component of conditioning in hematopoietic stem cell transplantation (HSCT). Due to the shift of donor preference to alternate donors, reliance on rabbit-ATG (rATG) has increased. Two different forms of rATG (Thymoglobuline® and Grafalon®) are available for clinical use but data to support the use of one over the other is sparse. We retrospectively analyzed data of 144 patients who underwent allogenic-HSCT for benign hematological conditions at our center, from August 2019 to August 2023. Of these, 87 received Grafalon® and 57 received Thymoglobuline®. The majority (77.7%) underwent HSCT for hemoglobinopathies and all received pre-transplant immunosuppression. Engraftment kinetics was similar in 2 cohorts. Six patients had primary graft failure (PGF). There was no difference in the incidence of PGF stratified by serotherapy. Overall survival(OS) for the cohort was 74.9%. Kaplan-Meier estimate of OSand EFSwas significantly better in Grafalon® group than Thymoglobuline® (84.4 ± 0.04% vs 64.1% ±0.065%) (p-value= 0.04%) and (84.4 ± 0.04% and 61.2%±0.065% (p-value = 0.01)). Extensive chronic GVHD was (14%) higher in Thymoglobuline® group and (2.3%) in Grafalon®. Immune reconstitution at day + 100 was not statistically different between the two groups. On univariate analysis, Thymoglobuline® serotherapy (OR (95% CI) =4.665 (1.2-18.04))was associated with increased risk of acute grade III-IV GvHD. In our study, Grafalon® tended to have better OS, decreased incidence of acute grade III-IV GvHD, and extensive cGVHD. There was no difference in engraftment kinetics, PGF, and immune reconstitution between 2 cohorts of serotherapy.

2.
J Pediatr Hematol Oncol ; 46(1): e91-e93, 2024 Jan 01.
Artigo em Inglês | MEDLINE | ID: mdl-38019672

RESUMO

Sirolimus (mammalian target of rapamycin inhibitor) is a potent immunosuppressive agent, used in patients receiving hematopoietic stem cell transplant (HSCT) for Graft vs Host disease prophylaxis. Compared to calcineurin inhibitors, sirolimus has no neurotoxicity or nephrotoxicity, but sirolimus causes dose-dependent thrombocytopenia, leukopenia, delayed wound healing, hyperlipidemia, and hypertriglyceridemia. Here we report a case of acute pancreatitis and diabetic ketoacidosis in a patient with sickle cell disease post haploidentical family donor HSCT which was managed conservatively without plasmapheresis. Based on our review of the literature, this is the first reported case of developing acute pancreatitis as an adverse effect of sirolimus-induced hypertriglyceridemia leading to diabetic ketoacidosis in a recipient of HSCT.


Assuntos
Anemia Falciforme , Diabetes Mellitus , Cetoacidose Diabética , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Hipertrigliceridemia , Pancreatite , Humanos , Sirolimo/uso terapêutico , Cetoacidose Diabética/induzido quimicamente , Cetoacidose Diabética/terapia , Cetoacidose Diabética/complicações , Doença Aguda , Pancreatite/induzido quimicamente , Pancreatite/terapia , Imunossupressores/efeitos adversos , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Hipertrigliceridemia/induzido quimicamente , Hipertrigliceridemia/terapia , Hipertrigliceridemia/complicações , Transplante de Células-Tronco/efeitos adversos , Anemia Falciforme/terapia , Anemia Falciforme/tratamento farmacológico , Diabetes Mellitus/induzido quimicamente , Diabetes Mellitus/tratamento farmacológico
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