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1.
Pediatr Transplant ; 26(4): e14239, 2022 06.
Artigo em Inglês | MEDLINE | ID: mdl-35122456

RESUMO

BACKGROUND: Infants are subjected to hematopoietic stem cell transplantation (HSCT) due to malignant and non-malignant diseases. However, specific data concerning the outcome and transplantation-related complications in infants, as a separate age group, are limited. Our aim was to evaluate the impact of infancy on the outcome, toxicity, and complications after HSCT. METHODS: We retrospectively analyzed data of 55 infants that underwent HSCT in our unit from May 1997 until February 2020, emphasizing on the probability of overall survival (OS) and the cumulative incidence (CI) of transplantation-related mortality (TRM) and complications. RESULTS: We report a probability of OS of 61%, a CI of TRM at day 100 and 365 post transplantation of 22% and 30%, respectively, and additionally a CI of graft failure, acute graft-versus-host disease (GvHD), and infectious complications, 18%, 44%, and 39%, respectively. No statistically significant association was detected between the above mentioned parameters and diagnosis, the use of myeloablative or non-myeloablative/reduced toxicity conditioning regimens or the type of donor. CONCLUSIONS: We conclude that HSCT in infancy is associated with significant mortality and morbidity. This is possibly attributed to endogenous, age-related factors. More specifically, infants may be at a higher risk of toxicities due to the immaturity of developing vital organs and the deficiency of the newly adopted immune system that predisposes them to infectious complications. The development of GvHD further augments the danger of infections, in a potential vice-versa relationship. Moreover, there are few data on pharmacokinetics of chemotherapy agents, making safe and efficacious drug administration hard.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Doença Enxerto-Hospedeiro/epidemiologia , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Morbidade , Recidiva Local de Neoplasia , Estudos Retrospectivos , Condicionamento Pré-Transplante/efeitos adversos
3.
J Pediatr Hematol Oncol ; 41(8): 612-617, 2019 11.
Artigo em Inglês | MEDLINE | ID: mdl-31259830

RESUMO

Prognostic refinement in Fanconi anemia (FA) is needed, especially when considering allogeneic hematopoietic stem cell transplantation (HCT). We studied 20 children with FA and bone marrow failure from a single center. According to Hôpital Saint-Louis risk classification for FA, patients were classified in stage A (no or mild cytopenia/dysplasia), B (single non-high-risk cytogenetic abnormality), C (severe cytopenia and/or significant dysplasia and/or high-risk cytogenetic abnormality), and D (myelodysplastic syndrome with excess of blasts/acute myeloid leukemia) in 4, 2, 13, and 0 cases, respectively. Nine patients received androgens +/- steroids, with a response rate of 30%, and 11 patients underwent HCT. Ten-year cumulative incidence (CI) of myelodysplastic syndrome/acute myeloid leukemia and overall survival (OS) were 21.9% and 45.3%, respectively, in the entire cohort, whereas cumulative incidence of transplantation-related mortality and OS were 27% and 63%, respectively, in patients who underwent HCT. Patients with significant dysplasia at diagnosis (stages C and D) had significantly shorter OS post-HCT as compared with patients without dysplasia. All patients in stages C and D at diagnosis or during evolution died from their disease. HCT in recent years was associated with more favorable outcomes. Larger cohorts could validate homogenous reporting of risk and help decision-making, particularly for HCT.


Assuntos
Anemia de Fanconi , Leucemia Mieloide Aguda , Síndromes Mielodisplásicas , Adolescente , Androgênios/administração & dosagem , Criança , Pré-Escolar , Intervalo Livre de Doença , Anemia de Fanconi/diagnóstico , Anemia de Fanconi/tratamento farmacológico , Anemia de Fanconi/genética , Anemia de Fanconi/mortalidade , Feminino , Humanos , Lactente , Leucemia Mieloide Aguda/diagnóstico , Leucemia Mieloide Aguda/tratamento farmacológico , Leucemia Mieloide Aguda/genética , Leucemia Mieloide Aguda/mortalidade , Masculino , Síndromes Mielodisplásicas/diagnóstico , Síndromes Mielodisplásicas/tratamento farmacológico , Síndromes Mielodisplásicas/genética , Síndromes Mielodisplásicas/mortalidade , Estudos Retrospectivos , Medição de Risco , Taxa de Sobrevida
4.
Hematology ; 21(1): 34-41, 2016 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-26218077

RESUMO

OBJECTIVES: The demethylating factor 5-Azacytidine (5-AZA) improves survival of patients with myelodysplastic syndromes (MDS) in randomized control trials but the results in 'real life' are controversial. METHODS: In this retrospective study, we evaluated the outcome of 56 high-risk MDS patients who were treated with 5-AZA between 2005 and 2013. 5-AZA was administered in an outpatient basis at a dose 75 mg/m(2) s.c. with the following schedule: 5 days on/weekend off/2 days on (5/2/2). RESULTS: The overall response rate (ORR) was 50%; 21.2% patients achieved complete response (CR), 3.8% partial response (PR), and 25% hematologic improvement (HI); 34.6% had stable disease (SD) and 15.4% showed progressive disease (PD). The estimated median event free survival (EFS) and overall survival (OS) were 11 and 17 months, respectively. Interestingly, the estimated time to acute myeloid leukemia transformation was 30 months, which refers to patients who responded to AZA treatment or remained stable. Patients who responded to the 5-AZA achieving CR, PR, and HI had better EFS and OS compared to the patients who had SD or PD. In addition, Δ WHO Classification-based Prognostic Score System (ΔWPSS), which represents the improvement of WPSS risk group before and after treatment, was associated with significantly improved OS and better EFS. Finally, the response to treatment was not associated with the expression of p53. CONCLUSIONS: In conclusion, 5-AZA is an effective treatment for high-risk MDS. Improved OS and EFS were found mainly in patients who responded to the treatment while ΔWPSS seems to represent a promising future prognostic tool.


Assuntos
Antimetabólitos Antineoplásicos/uso terapêutico , Azacitidina/uso terapêutico , Leucemia Mieloide Aguda/tratamento farmacológico , Síndromes Mielodisplásicas/tratamento farmacológico , Proteína Supressora de Tumor p53/genética , Idoso , Idoso de 80 Anos ou mais , Esquema de Medicação , Feminino , Expressão Gênica , Humanos , Leucemia Mieloide Aguda/diagnóstico , Leucemia Mieloide Aguda/etiologia , Leucemia Mieloide Aguda/mortalidade , Masculino , Pessoa de Meia-Idade , Síndromes Mielodisplásicas/complicações , Síndromes Mielodisplásicas/diagnóstico , Síndromes Mielodisplásicas/mortalidade , Prognóstico , Indução de Remissão , Estudos Retrospectivos , Medição de Risco , Análise de Sobrevida , Resultado do Tratamento
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