In total, 17 pediatric patients with hematologic malignancies (n ¼ 14) and Fanconi anemia (FA) (n ¼ 3) underwent haploidentical SCT with T-cell depletion. The patients were conditioned with reduced-intensity regimens, and CYA was used for GVHD prophylaxis. Successful engraftment occurred in 16 patients (94%). One patient failed to achieve a primary engraftment. Another patient rejected the first SCT after 10 weeks and had a successful second transplant. Of all engrafted patients, only one developed severe acute GVHD. Ten patients were alive at a median follow-up of 18 months (range, 5-62 months). The 5-years' OS was 53.8%. The three patients with FA are currently well with full-donor chimerism at 16, 6 and 5 months post transplant, respectively. The OS of 14 patients with high-risk hematologic malignancies was 47.6%. Three patients died as a result of post transplant leukemia relapse. CMV infection, GVHD and organ injury were other causes of mortality. Haploidentical SCT was found to be an alternative feasible treatment in Uruguay for patients who need allogenic transplantation but lack an HLA-identical family donor. It should be considered as an early option in FA patients before transformation or significant exposure to blood products.
Objective Identification of imaging prognostic parameters for early therapy personalisation to reduce treatment-related morbidity in paediatric Hodgkin lymphoma (HL). Our aim was to evaluate quantitative markers from baseline 2-[18F]fluoro-2-deoxy-d-glucose PET/CT as prognostic factors for treatment outcomes. Another goal was assessing the prognostic value of Deauville score at interim PET/CT. Methods Twenty-one patients were prospectively enrolled. Median age was 12 years (range 6–17); 13 were female. Patients underwent PET/CT for disease staging (bPET), at the end of two cycles of chemotherapy (iPET) and after chemotherapy. A total of 173 lesions were segmented from bPET. We calculated 51 texture features for each lesion. Total metabolic tumour volume and total lesion glycolysis from bPET were calculated for response prediction at iPET. Univariate and multivariate analyses were used for optimal cut-off values to separate responders at iPET according to the Deauville score. Results We identified four texture features as possible independent predictors of treatment outcomes at iPET. The areas under the ROC for univariate analysis were 0.89 (95% CI, 0.75–1), 0.82 (95% CI, 0.64–1), 0.79 (95% CI, 0.59–0.99) and 0.89 (95% CI, 0.75–1). The survival curves for patients assigned Deauville scores 1, 2, 3 and X were different from those assigned a score 4, with 4-year progression free-survival (PFS) rates of 85 versus 29%, respectively (P = 0.05). Conclusions We found four textural features as candidates for predicting early response to chemotherapy in paediatric patients with HL. The Deauville score at iPET was useful for differentiating PFS rates.
We evaluated the outcome of 71 children with de novo acute myeloid leukemia enrolled in 2 consecutive protocols in the main pediatric hospital in Uruguay. In the LAM97 protocol (n=34), patients received, as consolidation, autologous or allogeneic hematopoietic stem cell transplantation (HSCT), depending on the availability or not of a matched sibling donor. In the LAM08 protocol (n=37), patients were stratified into risk groups, autologous HSCT was abandoned, and allogeneic HSCT was limited to intermediate-risk patients with matched sibling donor and to all patients who fulfilled the high-risk criteria. Complete remission was achieved in 91% and 92% of patients in LAM97 and LAM08, respectively. Deaths in complete remission were 9.6% and 17.6%, respectively. The incidence of relapse was significantly higher in LAM97, 35.4%, versus 12.5% in LAM08. The 5-year event-free survival and overall survival were 50.0% and 55.9% in LAM97 and 59.9% and 64.8% in LAM08. The 5-year overall survival rates in each of the risk groups were 85.7% and 100% for low risk, 50.0% and 61.2% for intermediate risk, and 42.9% and 50.0% for high risk in LAM97 and LAM08 protocols, respectively. Survival has improved over the last 2 decades, and results are comparable to those published in Europe and North America.
Introducción: El neuroblastoma es el tumor sólido extracraneal más frecuente en niños. Aproximadamente el 50 % de los pacientes son clasificados como de alto riesgo, con base en características clínicas, biológicas e histológicas. Objetivo: Describir a la población asistida em el Centro Hemato-Oncológíco Pediátrico (CHOP) del Centro Hospitalario Pereira Rossell (CHPR) con diagnóstico de neuroblastoma de alto riesgo, su tratamiento y sobrevida. Método: Estudio descriptivo y retrospectivo de todos los pacientes con neuroblastoma de alto riesgo diagnosticados en el CHOP entre el 2001 y el 2015. En el CHOP se ubica el Registro Nacional de Cáncer Pediátrico, así como también el Archivo de Historias Clínicas de todos los pacientes. Los datos son recolectados y analizados por el sector de estadística. Resultados: Se diagnosticaron 35 pacientes, de los cuales 20 (57%) eran varones con mediana de edad de 36,6 meses (5-93), localización suprarrenal 23 (66%) y 100% estadio IV. Metástasis, médula ósea y hueso: 27 (71%). Treinta y tres pacientes recibieron autotransplante de progenitores hematopoyéticos (TPH) (94%). Estatus previo a TPH, remisión completa: 19 (58 %), remisión parcial: 14 (42%). Mortalidad relacionada al tratamiento: 15 % y de recaídas: 68 %. Mediana de tiempo de recaída: 15 meses (3-52). La probabilidad de sobrevida global y sobrevida libre de eventos a 5 años fue de 37,8% ± 8,4 y 23,8% ± 7,3 (mediana de seguimiento 40 meses). Conclusión: A pesar del tratamiento intensivo y de las medidas de soporte adecuadas, el pronóstico en los niños con neuroblastoma de alto riesgo sigue siendo pobre en nuestro país. Es necesario incorporar nuevas estrategias terapéuticas aún no disponibles en nuestro medio.
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