Results of intensive chemotherapy in children with juvenile chronic myelomonocytic leukemia: A pilot study

Heredia, Cristina Díaz de; Ortega, Juan José López; Coll, María T.; Bastida, Pilar; Olivé, T

doi:10.1002/(sici)1096-911x(199812)31:6<516::aid-mpo9>3.3.co;2-h

Cited by 4 publications

(4 citation statements)

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“…Similar to previous studies, we show that pre‐HCT therapy does not yield improvements for every JMML patient . However, by assessing for molecular responses, we were able to identify a subset of patients who experienced molecular remissions and went on to have an excellent PFS after HCT.…”

Section: Discussionsupporting

confidence: 82%

Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia

Hecht

Meyer

Chehab

et al. 2019

Pediatric Blood & Cancer

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Background: Despite the intensity of hematopoietic stem cell transplantation (HCT), relapse remains the most common cause of death in juvenile myelomonocytic leukemia (JMML). In contrast to other leukemias where therapy is used to reduce leukemic burden prior to transplant, many patients with JMML proceed directly to HCT with active disease. The objective of this study was to elucidate whether pre-HCT therapy has an effect on the molecular burden of disease and how this affects outcome post-HCT.Procedure: Twenty-one patients with JMML who received pre-HCT therapy and were transplanted at UCSF were analyzed in this study. The mutant allele frequency of the driver mutation was assessed before and after pre-HCT therapy, using custom amplicon next-generation sequencing.Results: Of the 21 patients, seven patients (33%) responded to therapy with a significant reduction in their mutant allele frequency and were classified as molecular responders. Six of these patients received moderate-intensity chemotherapy, one patient received only azacitidine. The 5-year progression-free survival after HCT of molecular responders was 100% versus 61% for nonresponders (P = .12). Survival of molecular nonresponders was not improved by use of highintensity conditioning, but patients were salvaged if they experienced severe graft versus host disease. There were no baseline clinical characteristics that were associated with response to pre-HCT therapy. Conclusions:Despite the myelodysplastic nature of JMML, patients treated with pre-HCT therapy can achieve molecular remissions. These patients experienced a trend toward improved outcomes post-HCT. Importantly, molecular testing can be helpful to distinguish between responders and nonresponders and should become an integral part of clinical care.

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Section: Discussionsupporting

confidence: 82%

Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia

Hecht

Meyer

Chehab

et al. 2019

Pediatric Blood & Cancer

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“…Most approaches to intensive chemotherapy are derived from treatment protocols for AML. Clinical remissions and long‐term survival after AML‐type combination therapy have been reported in small series (reviewed in Niemeyer & Locatelli, 2006; Chan et al , 1987; Diaz de Heredia et al , 1998). Study CCG 2891 of the Children’s Cancer Study Group for treatment of childhood AML, MDS and JMML included 13 children with JMML (Woods et al , 2002).…”

Section: Molecular Pathogenesis Of Jmmlmentioning

confidence: 99%

Paediatric myelodysplastic syndromes and juvenile myelomonocytic leukaemia: molecular classification and treatment options

2008

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SummaryMyelodysplastic syndromes (MDS) and the mixed myelodysplastic/myeloproliferative disorder juvenile myelomonocytic leukaemia (JMML) are rare haematopoietic stem cell diseases in children. While MDS-initiating events remain largely obscure, a growing body of clinical, genetic and laboratory evidence suggests that JMML is, at least in part, caused by aberrant signal transduction resulting from mutations of components of the RAS signalling pathway. To date, haematopoietic stem cell transplantation cures more than half of children diagnosed with MDS or JMML. Research on genetic conditions predisposing to MDS in young age, such as inherited syndromes with bone marrow failure, may present important insights into MDS pathogenesis.

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“…tG y MtX: tratamiento de mantención con metotrexato y tioguanina, SdA: síndrome diarreico agudo, VMI: ventilación mecánica invasiva, 1RC: primera remisión completa, Ph (+): cromosoma filadelfia positivo, ESPhALL: protocolo de tratamiento de la leucemia linfoblástica aguda con cromosoma filadelfia positivo en el grupo PINDA. LMC 1FC: leucemia mieloide crónica en primera fase crónica, LMMJ: leucemia mielomonocítica juvenil, Vall d'Hebron: protocolo de tratamiento 16 , LMA PIndA 2006: protocolo de tratamiento de la leucemia mieloide aguda del PIndA versión 2006. SBOR: síndrome bronquial obstructivo recurrente.…”

Section: Resultsunclassified

Donación dirigida de sangre de Cordón Umbilical de Hermano Compatible en el Sistema de Salud Público de Chile

et al. 2020

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Introducción: La sangre de cordón umbilical (SCU) como fuente para trasplante de células progenitoras hematopoyéticas (TPH) está bien establecida. Internacionalmente, menos del 10% de los TPH de SCU corresponde a donantes hermanos compatibles. Dentro de la red del Programa Infantil Nacional de Drogas Antineoplásicas (PINDA), existe desde enero 2004 un programa de donación dirigida de SCU para TPH.Pacientes y Método: Se diseñó un estudio observacional, retrospectivo, descriptivo, se revisaron el número y características de las unidades de SCU recolectadas en el PINDA y el número, características y evolución de los pacientes trasplantados con esas unidades entre enero de 2004 y octubre de 2018.Resultados: Sesenta unidades de SCU han sido recolectadas, de ellas 55 con registro completo. La mediana de volumen de las unidades almacenadas fue 74,8 ml (30,0-170,8), la mediana de células nucleadas totales 7,6 x 10e8 (2,0-21,1), mediana de células CD34+ 1,6 x 10e6 (0,2-11,6). Cuatro pacientes con leucemias de alto riesgo fueron trasplantados; mediana de seguimiento es de 8 años. Todos desarrollaron complicaciones severas post TPH, uno de ellos falleció de recaída y los tres actualmente vivos presentan un Karnofsky/Lansky 100%.Conclusión: El programa ha permitido el trasplante de 4 pacientes que de otro modo no habrían tenido acceso a un donante. Este programa de donación dirigida puede ser considerado una primera etapa para el desarrollo de un banco público de sangre de cordón umbilical en Chile.

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Results of intensive chemotherapy in children with juvenile chronic myelomonocytic leukemia: A pilot study

Cited by 4 publications

References 0 publications

Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia

Molecular assessment of pretransplant chemotherapy in the treatment of juvenile myelomonocytic leukemia

Paediatric myelodysplastic syndromes and juvenile myelomonocytic leukaemia: molecular classification and treatment options

Donación dirigida de sangre de Cordón Umbilical de Hermano Compatible en el Sistema de Salud Público de Chile

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