Allogeneic hematopoietic stem cell transplantation in pediatric myelodysplastic syndromes: A multicenter experience from Argentina

Basquiera, Ana Lisa; Silvia, Pizzi; Correas, Agustín González; Longo, Pablo G.; Goldman, Wanda C.; Prates, María Virginia; Formisano, Sandra; Kusminisky, Gustavo; Feldman, Leonardo; Berretta, Adriana; Garcı́a, Juan José; Staciuk, Raquel

doi:10.1002/pbc.25238

Cited by 11 publications

(17 citation statements)

References 23 publications

(80 reference statements)

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“…The conditioning regimens in our study were variable; all were myeloablative and mainly busulfan based. In contrast to a multicenter report from Argentina by Basquiera et al . evaluating allogeneic HSCT in pediatric MDS and showing that UCB transplantation was associated with lower OS, the graft source in our study did not have a significant influence on outcome.…”

Section: Discussioncontrasting

confidence: 99%

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Outcomes of children, adolescents, and young adults following allogeneic stem cell transplantation for secondary acute myeloid leukemia and myelodysplastic syndromes—The MD Anderson Cancer Center experience

Maher

Silva

et al. 2017

Pediatric Transplantation

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We conducted a retrospective analysis of outcomes for children and young adults with sAML/sMDS who underwent HSCT at our institution. Thirty-two patients (median age 20 years) with sAML (n=24) and sMDS (n=8) received HSCT between 1990 and 2013. The median time from sAML/sMDS diagnosis to HSCT was 4.1 months (range: 1.2-27.2 months). The transplant regimens were primarily busulfan based (n=19). BM was the primary donor source (n=15). Eleven recipients were transplanted with residual disease. At a median follow-up of 62.3 months (range: 0.4-250.9 months), 14 patients had disease recurrence. Acute GVHD, grade III/IV, occurred in three patients. Causes of death were as follows: disease relapse (n=12), infection (n=2), pneumonia (n=1), pulmonary hemorrhage (n=1), acute GVHD (n=1), and graft failure (n=1). A PS of ≥90% at the time of HSCT had a significant impact on PFS (P=.02). Patients achieving pretransplant primary CR (n=8) and those with sMDS and RA (n=6) had prolonged PFS (P=.04). On multivariate analysis, shorter time to transplantation (≤6 months from diagnosis of sAML/sMDS) was associated with superior OS (P=.0018) and PFS (P=.0005).

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Section: Discussioncontrasting

confidence: 99%

“…There are few published reports on the outcome of allogeneic HSCT in CAYA with sAML/sMDS . Most of the previous reports on outcome of allogeneic SCT for sAML/sMDS focus on the adult population, with median ages between 35 and 40 years .…”

Section: Introductionmentioning

confidence: 99%

Outcomes of children, adolescents, and young adults following allogeneic stem cell transplantation for secondary acute myeloid leukemia and myelodysplastic syndromes—The MD Anderson Cancer Center experience

Maher

Silva

et al. 2017

Pediatric Transplantation

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“…20 Further, the EWOG-MDS group showed the 5-year OS probability in 97 children with advanced MDS or MDR-AML receiving HSCT from an HLA-matched sibling (MSD) or an unrelated donor (UD) following a preparative regimen with busulfan, cyclophosphamide, and melphalan was 63%, while the 5-year cumulative incidence of transplantation-related mortality (TRM) and relapse were both 21%. 19 In our study, we found that the OS and DFS were comparable to previous clinical studies [19][20][21] [20][21][22] Grade III aGVHD comprised a major proportion of events in our study. However, these events were controllable and did not affect survival in our study.…”

Section: Discussionsupporting

confidence: 88%

Unmanipulated haploidentical hematopoietic stem cell transplantation for children with myelodysplastic syndrome

Suo¹,

Wang²,

Xue

et al. 2020

Pediatric Transplantation

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Myelodysplastic syndrome (MDS) is a rare childhood disease, with an annual incidence of 1 to 4 cases per million. This accounts for less than 5% of all pediatric hematological malignancies. 1,2 Low-grade MDS [refractory cytopenia of childhood (RCC)] and advanced MDS [refractory anemia with excess blasts (RAEB) and RAEB in transformation (RAEB-T)] are the most recent pediatric MDS classifications. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curative option currently available for childhood MDS, especially

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“…The progression to secondary AML is variable, from slow period of several months to sudden transformation (7). It occurs in about 30% of cases of pediatric MDS, usually up to 2 years from diagnosis (13). In contrast to adult MDS, significance of subclassification into RAEB1 and RAEB 2 is not clear (14,15).…”

Section: Classificationsmentioning

confidence: 99%

The Differencies in Adult and Pediatric Myelodysplastic Syndrome: A Review

Vasekova

2016

Acta Medica Martiniana

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Myelodysplastic syndrome (MDS) represent very heterogenous group of clonal stem cell bone marrow disorders with ineffective haematopoesis leading to cytopenias in peripheral blood and increased risk of blastic transformation and evolution of acute myeloid leukemia. MDS is a disease of older age mostly, in children it seems to be very rare. There are several significant morphological, cytogenetic and prognostic differencies of the disease in adults and in children. Adult MDS patients most commonly manifest with symptoms of anemia, bleeding and infection are uncommon. In childhood, MDS manifests predominantly by neutropenia and thrombocytopenia. In addition, some pediatric MDS patients present also with constitutional disease’s signs and symptoms. Early and correct diagnosis in both age groups is essential for the choice of appropriate therapy and also for next life of patients. However, the diagnosis of MDS is challenging, complex and requiring close correlation of clinical symptoms, laboratory parameters and standardized examination of BM biopsies. The authors present an overview focused on biology of MDS in adults and children, on the differences in the incidence, clinical presentation and treatment. They summarize the possibilities and limits of histopathological diagnosis and differential diagnosis of the disease in different age groups. A major problem in the morphological diagnosis of MDS remains the determination, whether the myelodysplasia is due to clonal disorder. It might result also from some other factors, as significant dysplasia can also occur in reactive conditions, and vice versa, only discrete dysplasia is sometimes observed in MDS patients. Although histomorphological and immunohistochemical analysis of BM biopsy is invasive and time-consuming examination, it has its value in the diagnosis, differential diagnosis and evaluation of therapeutic effect.

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Allogeneic hematopoietic stem cell transplantation in pediatric myelodysplastic syndromes: A multicenter experience from Argentina

Abstract: In our series, more than half of the patients achieved long term OS with AHSCT. Less toxic conditioning regimens or more intensive GVHD prophylaxis could lead to better results in some children.

Cited by 11 publications

References 23 publications

Outcomes of children, adolescents, and young adults following allogeneic stem cell transplantation for secondary acute myeloid leukemia and myelodysplastic syndromes—The MD Anderson Cancer Center experience

Outcomes of children, adolescents, and young adults following allogeneic stem cell transplantation for secondary acute myeloid leukemia and myelodysplastic syndromes—The MD Anderson Cancer Center experience

Unmanipulated haploidentical hematopoietic stem cell transplantation for children with myelodysplastic syndrome

The Differencies in Adult and Pediatric Myelodysplastic Syndrome: A Review

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