How I treat myelodysplastic syndromes of childhood

Pediatric myelodysplastic syndromes (MDSs) are a heterogeneous group of clonal disorders with an annual incidence of 1 to 4 cases per million, accounting for less than 5% of childhood hematologic malignancies. MDSs in children often occur in the context of inherited bone marrow failure syndromes, which represent a peculiarity of myelodysplasia diagnosed in pediatric patients. Moreover, germ line syndromes predisposing individuals to develop MDS or acute myeloid leukemia have recently been identified, such as those caused by mutations in GATA2, ETV6, SRP72, and SAMD9/SAMD9-L. Refractory cytopenia of childhood (RCC) is the most frequent pediatric MDS variant, and it has specific histopathologic features. Allogeneic hematopoietic stem cell transplantation (HSCT) is the treatment of choice for many children with MDSs and is routinely offered to all patients with MDS with excess of blasts, to those with MDS secondary to previously administered chemoradiotherapy, and to those with RCC associated with monosomy 7, complex karyotype, severe neutropenia, or transfusion dependence. Immune-suppressive therapy may be a treatment option for RCC patients with hypocellular bone marrow and the absence of monosomy 7 or a complex karyotype, although the response rate is lower than that observed in severe aplastic anemia, and a relevant proportion of these patients will subsequently need HSCT for either nonresponse or relapse.
Source: Blood - Category: Hematology Authors: Tags: Pediatric Hematology, Transplantation, How I Treat, Free Research Articles, Myeloid Neoplasia Source Type: research

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Conclusions: the frequencies of infections in maternal-fetal health were considered low, highlighting on syphilis and the presence for sickle cell trait.Resumo Objetivos: estimar a taxa de detec ção de patologias da TPN em papel de filtro nas regiões Sul e Sudoeste do Estado da Bahia, bem como delinear o perfil epidemiológico dessas gestantes, calcular e estimar a taxa de adesão e abrangência de cobertura do Programa. Métodos: estudo descritivo, de agosto 2013 a agosto de 2015, de d ados obtidos do Labimuno/ICS/UFBA. Resultados: foram incluídas 64.743 gestantes; as m&e...
Source: Revista Brasileira de Saude Materno Infantil - Category: Midwifery Source Type: research
Publication date: Available online 15 September 2019Source: Advances in Biological RegulationAuthor(s): Samuel Gusscott, Francesco Tamiro, Vincenzo Giambra, Andrew P. WengAbstractT-cell acute lymphoblastic leukemia (T-ALL) is an aggressive cancer, characterized by an uncontrolled expansion and accumulation of T-cell progenitors. During leukemic progression, immature T cells grow abnormally and occupy the bone marrow compartment, thereby interfering with the production of normal blood cells. Pediatric T-ALL is curable with intensive chemotherapy, but there are significant, long-term side effects and ∼20% of patients suf...
Source: Advances in Biological Regulation - Category: Biology Source Type: research
In this study, we investigated the influence of structure of extracellular matrix-mimicking electrospun poly (l-lactic acid) (PLLA) fibrous scaffolds and incorporated growth factors (bone morphogenetic protein2 (BMP2) and platelet-derived growth factor (PDGF)) (GFs) on in vitro differentiation of human mesenchymal stem cells (hMSCs) cultured onto the scaffolds. Furthermore, Cargo GFs were first encapsulated into coacervate (Coa). Coa-coated nano-sized PLLA (Coa-nPLLA) exhibited relatively higher coating efficacy, than Coa-coated micro-sized PLLA (Coa-mPLLA) due to large mesh hole area of mPLLA. Consequently, a faster GF re...
Source: Journal of Industrial and Engineering Chemistry - Category: Chemistry Source Type: research
Condition:   Multiple Myeloma Interventions:   Drug: Belantamab mafodotin;   Drug: Bortezomib;   Drug: Lenalidomide;   Drug: Dexamethasone Sponsor:   GlaxoSmithKline Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Condition:   Leukemia, Myeloid, Acute Interventions:   Drug: Pevonedistat;   Drug: Azacitidine Sponsors:   PETHEMA Foundation;   Millennium Pharmaceuticals, Inc.;   Dynamic Science S.L. Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Conditions:   Advanced Malignant Solid Neoplasm;   Caregiver;   Metastatic Malignant Solid Neoplasm;   Recurrent Leukemia;   Recurrent Lymphoma;   Recurrent Malignant Solid Neoplasm;   Recurrent Plasma Cell Myeloma Intervention:   Other: Questionnaire Administration Sponsors:   M.D. Anderson Cancer Center;   National Cancer Institute (NCI) Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Myelodysplastic syndromes (MDS) are clonal diseases defined by clinical, morphologic, and genetic features often shared by related myeloid disorders. The diagnostic boundaries between these diseases can be arbitrary and not necessarily reflective of underlying disease biology or outcomes. In practice, measures that distinguish MDS from related disorders may be difficult to quantify and can vary as disease progression occurs. Patients may harbor findings that are not consistent with a single diagnostic category. Several overlap disorders have been formally described, such as the myelodysplastic/myeloproliferative neoplasms ...
Source: Blood - Category: Hematology Authors: Tags: Hematopoiesis and Stem Cells, Myeloid Neoplasia, Review Articles, Review Series, Clinical Trials and Observations Source Type: research
This study may open up new potential therapeutic avenues for the treatment of patients with chronic infection, inflammatory diseases, and cancer.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - Category: Hematology Authors: Tags: 506. Hematopoiesis and Stem Cells: Microenvironment, Cell Adhesion, and Stromal Stem Cells Source Type: research
Acquired aplastic anemia and inherited bone marrow failure syndromes both present with pancytopenia and must be distinguished because they have differences in treatment decisions and continued monitoring requirements. Advances in the genetic interrogation of patient samples have led to identification of inherited germline diseases and appreciation that patients with inherited bone marrow failure disorders may be normal in appearance with few expected clinical clues. Somatic mutations in aplastic anemia may have prognostic value. Hematopoietic stem cells from inherited marrow failure diseases can correct the proliferative d...
Source: Hematology/Oncology Clinics of North America - Category: Cancer & Oncology Authors: Source Type: research
Authors: West AH, Churpek JE Abstract Patients with inherited bone marrow failure syndromes (IBMFSs) classically present with specific patterns of cytopenias along with congenital anomalies and/or other physical features that are often recognizable early in life. However, increasing application of genomic sequencing and clinical awareness of subtle disease presentations have led to the recognition of IBMFS in pediatric and adult populations more frequently than previously realized, such as those with early onset myelodysplastic syndrome (MDS). Given the well-defined differences in clinical management needs and outc...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
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