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RESUMO O Consenso Brasileiro de Nutrição em Transplante de Células-Tronco Hematopoiéticas: crianças e adolescentes foi elaborado com a participação de nutricionistas, médicos nutrólogos e médicos hematologistas pediátricos de 10 centros brasileiros que são referência em transplante de células-tronco hematopoiéticas. O objetivo foi salientar a importância do estado nutricional e da composição corporal durante o tratamento, bem como as principais características relacionadas à avaliação nutricional do paciente. As intenções, ao se estabelecer o consenso, foram aprimorar e padronizar a terapia nutricional durante o transplante de células-tronco hematopoiéticas. O consenso foi aprovado pela Sociedade Brasileira de Transplante de Médula Óssea.

ABSTRACT The Brazilian Nutritional Consensus in Hematopoietic Stem Cell Transplantation: Children and Adolescents was developed by dietitians, physicians, and pediatric hematologists from 10 Brazilian reference centers in hematopoietic stem cell transplantation. The aim was to emphasize the importance of nutritional status and body composition during treatment, as well as the main characteristics related to patient´s nutritional assessment. This consensus is intended to improve and standardize nutrition therapy during hematopoietic stem cell transplantation. The consensus was approved by the Brazilian Society of Bone Marrow Transplantation.

https://doi.org/10.31744/einstein_journal/2021ae5254

Associação Brasileira de Hematologia, Hemoterapia e Terapia Celular Consensus on genetically modified cells. II: CAR-T cell therapy for patients with CD19+ acute lymphoblastic leukemia
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Seber, Adriana

; Castro Junior, Claudio Galvãode de

; Kerbauy, Lucila N

; Hirayama, Alexandre V

; Bonfim, Carmem

; Fernandes, Juliana Folloni

; Souza, Mair

; Schafell, Rony

; Nabhan, Samir

; Loggetto, Sandra Regina

; Simões, Belinda Pinto

; Rocha, Vanderson

; Lima, Marcos de

; Guerino-Cunha, Renato L

; Bittencourt, Henrique

Hematology, Transfusion and Cell Therapy

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2021, Volume 43 Suppl. 2 Pages 13 - 21

Abstract: En | Text: En | PDF: En

ABSTRACT Chimeric antigen receptor T (CAR-T) cell therapy is a novel therapeutic modality for acute lymphoblastic leukemia (ALL) with robust outcomes in patients with refractory or relapsed disease. At the same time, CAR-T cell therapy is associated with unique and potentially fatal toxicities, such as cytokine release syndrome (CRS) and neurological toxicities (ICANS). This manuscript aims to provide a consensus of specialists in the fields of Hema- tology Oncology and Cellular Therapy to make recommendations on the current scenario of the use of CAR-T cells in patients with ALL.

https://doi.org/10.1016/j.htct.2021.09.002

Imaging clinical case
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Xavier, Fernando Silva

; Duarte, Márcio Luís

; Seber, Adriana

Nascer e Crescer

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dec 2020, Volume 29 N. 4 Pages 222 - 224

Abstract: Pt | Text: Pt | PDF: Pt

https://doi.org/10.25753/birthgrowthmj.v29.i4.19737 65 downloads

A consensus document for the clinical management of invasive fungal diseases in pediatric patients with hematologic cancer and/or undergoing hematopoietic stem cell transplantation in Brazilian medical centers
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Carlesse, Fabianne

; Daudt, Liane Esteves

; Seber, Adriana

; Dutra, Álvaro Pimenta

; Melo, Analy Salles de Azevedo

; Simões, Belinda

; Macedo, Carla Renata Donato

; Bonfim, Carmem

; Benites, Eliana

; Gregianin, Lauro

; Batista, Marjorie Vieira

; Abramczyk, Marcelo

; Tostes, Vivian

; Lederman, Henrique Manoel

; Lee, Maria Lúcia de Martino

; Loggetto, Sandra

; Castro Junior, Cláudio Galvão de

; Colombo, Arnaldo Lopes

Brazilian Journal of Infectious Diseases

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dec 2019, Volume 23 N. 6 Pages 395 - 409

Abstract: En | Text: En | PDF: En

ABSTRACT In the present paper we summarize the suggestions of a multidisciplinary group including experts in pediatric oncology and infectious diseases who reviewed the medical literature to elaborate a consensus document (CD) for the diagnosis and clinical management of invasive fungal diseases (IFDs) in children with hematologic cancer and those who underwent hematopoietic stem-cell transplantation. All major multicenter studies designed to characterize the epidemiology of IFDs in children with cancer, as well as all randomized clinical trials addressing empirical and targeted antifungal therapy were reviewed. In the absence of randomized clinical trials, the best evidence available to support the recommendations were selected. Algorithms for early diagnosis and best clinical management of IFDs are also presented. This document summarizes practical recommendations that will certainly help pediatricians to best treat their patients suffering of invasive fungal diseases.

https://doi.org/10.1016/j.bjid.2019.09.005 894 downloads

The influence of cell concentration at cryopreservation on neutrophil engraftment after autologous peripheral blood stem cell transplantation
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Félix, Olga Margareth Wanderley de Oliveira

; Tunes, Gisela

; Ginani, Valéria Cortez

; Simões, Paulo César

; Barros, Daniele Porto

; Delbuono, Elizabete

; Alves, Maria Teresa de Seixas

; Petrilli, Antonio Sérgio

; Lee, Maria Lúcia de Martino

; Gouveia, Roseane Vasconcelos

; Zecchin, Victor Gottardello

; Seber, Adriana

Hematology, Transfusion and Cell Therapy

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sep 2018, Volume 40 N. 3 Pages 233 - 239

Abstract: En | Text: En | PDF: En

ABSTRACT Background: Peripheral blood stem cell concentrations are traditionally adjusted to 20–40 × 106 leukocytes/mL prior to freezing. This low cell concentration at cryopreservation implies larger volumes with more dimethyl sulfoxide being used, and higher cost and toxicity at the time of transplant. Higher cell concentrations have been reported but this is not widely accepted. Moreover, the influence of cell concentration on engraftment has not been well documented. Therefore, this study retrospectively analyzed the influence of peripheral blood stem cell concentration at freezing on engraftment after autologous hematopoietic stem cell transplantation. Method: Leukapheresis products were plasma-depleted and cryopreserved with 5% dimethyl sulfoxide, 6% hydroxyethylamide solution and 4% albumin in a −80 °C freezer. Individual patient data from hospital records were reviewed. Results: Fifty consecutive patients with oncological diseases underwent 88 leukaphereses. Median age was six years (range: 1–32 years) and median weight was 19 kg (range: 8–94 kg). Median leukocyte concentration was 109 × 106/mL at collection and 359 × 106 (range: 58–676 × 106) at freezing with 78% viability (range: 53–95%); leukocyte recovery after thawing was 95% (range: 70–100%). In multivariate analysis, cell concentration (p-value = 0.001) had a negative impact on engraftment. Patients infused with bags frozen with <200 × 106 leukocytes/mL engrafted after a median of nine days (range: 8–12 days), 200–400 × 106 leukocytes/mL after 11 days (range: 9–20 days); 400–600 × 106 leukocytes/mL after 12 days (range: 8–19 days) and with cell concentrations >600 × 106 leukocytes/mL, engraftment was after 14 days (range: 13–22 days). Conclusion: In patients with adequate CD34 cell collections, total leukocyte concentrations of 282 × 106/mL, freezing with 5% dimethyl sulfoxide and 6% hydroxyethylamide solution without a controlled-rate freezer, and storing cells at −80 ºC yielded excellent engraftment. Further increases in cell concentration may delay engraftment, without affecting safety.

https://doi.org/10.1016/j.htct.2018.01.007 1034 downloads

Optimisation of a quantitative polymerase chain reaction-based strategy for the detection and quantification of human herpesvirus 6 DNA in patients undergoing allogeneic haematopoietic stem cell transplantation
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Ueda, Miriam YH

; Alvarenga, Paulo G

; Real, Juliana M

; Moreira, Eloisa de Sá

; Watanabe, Aripuanã

; Passos-Castilho, Ana Maria

; Vescovi, Matheus

; Novis, Yana

; Rocha, Vanderson

; Seber, Adriana

; Oliveira, Jose SR

; Rodrigues, Celso A

; Granato, Celso FH

Memórias do Instituto Oswaldo Cruz

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jun 2015, Volume 110 N. 4 Pages 461 - 467

Abstract: En | Text: En | PDF: En

Human herpesvirus 6 (HHV-6) may cause severe complications after haematopoietic stem cell transplantation (HSCT). Monitoring this virus and providing precise, rapid and early diagnosis of related clinical diseases, constitute essential measures to improve outcomes. A prospective survey on the incidence and clinical features of HHV-6 infections after HSCT has not yet been conducted in Brazilian patients and the impact of this infection on HSCT outcome remains unclear. A rapid test based on real-time quantitative polymerase chain reaction (qPCR) has been optimised to screen and quantify clinical samples for HHV-6. The detection step was based on reaction with TaqMan® hydrolysis probes. A set of previously described primers and probes have been tested to evaluate efficiency, sensitivity and reproducibility. The target efficiency range was 91.4% with linearity ranging from 10-106 copies/reaction and a limit of detection of five copies/reaction or 250 copies/mL of plasma. The qPCR assay developed in the present study was simple, rapid and sensitive, allowing the detection of a wide range of HHV-6 loads. In conclusion, this test may be useful as a practical tool to help elucidate the clinical relevance of HHV-6 infection and reactivation in different scenarios and to determine the need for surveillance.

https://doi.org/10.1590/0074-02760150004 1401 downloads

Shed some (sun)light on vitamin D deficiency
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Seber, Adriana

Revista Brasileira de Hematologia e Hemoterapia

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jun 2014, Volume 36 N. 3 Pages 167 - 168

Abstract: En | Text: En | PDF: En

https://doi.org/10.1016/j.bjhh.2014.03.017 1157 downloads

I Diretriz brasileira de cardio-oncologia pediátrica da Sociedade Brasileira de Cardiologia
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Seber, Adriana

; Miachon, Adriana Siviero

; Tanaka, Ana Cristina Sayuri

; Castro, Ângela Maria Spínola e

; Carvalho, Antônio Carlos

; Petrilli, Antônio Sergio

; Macedo, Carla Renata Donato Pacheco

; Nars, Clarissa Carvalho Fongaro

; Terzian, Claudia Naufel

; Castro Junior, Claudio Galvão de

; Santos, Cleusa Cavalcanti Lapa

; Guerra, Cristina Chaves dos Santos

; Silva, Dafne Cardoso Bourguignon da

; Bassi, Débora Ugayama

; Azeka, Estela

; Feitosa, Fabiana Aragão

; Hamamoto, Fernando

; Szarf, Gilberto

; Lederman, Henrique Manoel

; Rigon Junior, Humberto João

; Jatene, Ieda Biscegli

; Mota, Isabele Coelho Fonseca da

; Perrud, Jeferson Adriano

; Soares Junior, José

; Gutierrez, Julián Arango

; Perin, Juliana Pepe Marinho

; Soares, Juliana dos Santos

; Catani, Liane Hulle

; Tsai, Liliana Yu

; Vianna, Livia Cristina

; Paiva, Marcelo Goulart

; Santos, Marcelo José

; Ishigai, Marcia Marcelino de Souza

; Diógenes, Maria Suely Bezerra

; Alves, Maria Teresa de Seixas

; Piedade, Maria Tereza Castro

; Parreiras, Mariana

; Cypriano, Mônica

; Negrini, Nilce da Silva

; Campos Filho, Orlando

; Figueiredo, Paula Andrade

; Novaes, Paulo Eduardo

; Camargo, Paulo Roberto

; Maia, Priscila dos Santos

; Petrilli, Renata

; Arduini, Rodrigo Genaro

; Gouveia, Roseane Vasconcelos

; Teruya, Suzana Barbosa Miranda

; Moisés, Valdir Ambrosio

; Morais, Vera Lúcia Lins de

Arquivos Brasileiros de Cardiologia

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may 2013, Volume 100 N. 5 Pages 1 - 68

Abstract: Pt | Text: Pt | PDF: Pt

https://doi.org/10.5935/abc.2013S005 17336 downloads

Recommended screening and preventive practices for long-term survivors after hematopoietic cell transplantation
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Majhail, Navneet Singh

; Rizzo, James Douglas

; Lee, Stephanie Joi

; Aljurf, Mahmoud

; Atsuta, Yoshiko

; Bonfim, Carmem

; Burns, Linda Jean

; Chaudhri, Naeem

; Davies, Stella

; Okamoto, Shinichiro

; Seber, Adriana

; Socie, Gerard

; Szer, Jeff

; Lint, Maria Teresa Van

; Wingard, John Reid

; Tichelli, Andre

Revista Brasileira de Hematologia e Hemoterapia

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2012, Volume 34 N. 2 Pages 109 - 133

Abstract: Pt En | Text: Pt En | PDF: Pt | PDF: En

Os avanços na tecnologia do transplante de células hematopoéticas (TCH) e do tratamento de suporte levaram a melhoria na sobrevida a longo prazo após os TCH. Indicações emergentes de transplante, introdução de novas fontes de células (p.ex. sangue de cordão umbilical) e transplante de pacientes mais velhos utilizando regimes de condicionamento menos intensos também contribuíram para o aumento no número de sobreviventes após TCH. Estes sobreviventes estão sob risco de desenvolver complicações tardias devido a exposições e fatores de risco pré, peri e pós-transplante. Práticas recomendadas para a triagem e a prevenção de complicações em sobreviventes de TCH foram publicadas em 2006. Um grupo internacional de especialistas foi formado em 2011 para rever a literatura contemporânea e atualizar as recomendações, considerando as mudanças nas práticas de transplante e a aplicabilidade internacional destas recomendações. Esta revisão fornece as recomendações atualizadas para o diagnóstico precoce e práticas para prevenção de complicações aos sobreviventes de TCH autólogo e alogênico, adultos e crianças.

Advances in hematopoietic cell transplantation (HCT) technology and supportive care techniques have led to improvements in long-term survival after HCT. Emerging indications for transplantation, introduction of newer graft sources (e.g. umbilical cord blood) and transplantation of older patients using less intense conditioning regimens have also contributed to an increase in the number of HCT survivors. These survivors are at risk for developing late complications secondary to pre-, periand post-transplant exposures and risk-factors. Guidelines for screening and preventive practices for HCT survivors were published in 2006. An international group of transplant experts was convened in 2011 to review contemporary literature and update the recommendations while considering the changing practice of transplantation and international applicability of these guidelines. This review provides the updated recommendations for screening and preventive practices for pediatric and adult survivors of autologous and allogeneic HCT.

https://doi.org/10.5581/1516-8484.20120032 101668 downloads Cited 8 times in SciELO

10.

Indicações de transplante de células-tronco hematopoéticas em pediatria: consenso apresentado no I Encontro de Diretrizes Brasileiras em Transplante de Células-Tronco Hematopoéticas - Sociedade Brasileira de Transplante de Medula Óssea, Rio de Janeiro, 2009
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Seber, Adriana

; Bonfim, Carmem Maria S.

; Daudt, Liane E.

; Gouveia, Roseane V.

; Ginani, Valéria C.

; Mauad, Marcos

; Castro Jr, Claudio G.

Revista Brasileira de Hematologia e Hemoterapia

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2010, Volume 32 N. 3 Pages 225 - 239

Abstract: Pt En | Text: Pt En | PDF: Pt | PDF: En

A Sociedade Brasileira de Transplante de Medula (SBTMO) promoveu o I Encontro de Diretrizes do Transplante de Medula Óssea em 2009. Para revisão das indicações de transplante em Pediatria baseadas em evidências foi constituído grupo de trabalho com oncologistas e hematologistas com experiência em pediatria. Os artigos científicos foram cuidadosamente avaliados e, para cada doença, foram definidas as evidências para recomendação dos transplantes (de A a C) e a qualidade destas evidências (de 1 a 3). As recomendações incluem doenças hematológicas malignas e não malignas, tumores sólidos, imunodeficiências e doenças de depósito tratadas com transplantes de células-tronco hematopoéticas, quer autólogos, alogênicos de irmão HLA compatível ou não aparentados (doadores adultos ou sangue de cordão umbilical). Como não existem recomendações uniformemente aceitas em pediatria, não foram incluídas recomendações para transplantes de intensidade reduzida, com manipulação do enxerto e nem parcialmente compatíveis. É importante ressaltar que todas as indicações são baseadas no conhecimento atual e podem modificar-se com o tempo. Assim, esta revisão não deve ser utilizada para aplicação direta no cuidado do paciente sem levar em conta características da doença, do doador e fatores de risco do próprio paciente. Este trabalho não deve ainda ser utilizado como documento que limite o acesso do paciente ao transplante adequadamente indicado. Ressaltamos ainda, nesta revisão, diferenças entre transplantes em crianças e em adultos, com algumas recomendações específicas para os transplantes em pediatria.

The Brazilian Bone Marrow Transplant Society (SBTMO) held its First Meeting on Bone Marrow Transplant Guidelines in 2009. A working group of hematologists and oncologists with experience in pediatrics was formed to review evidence-based indications for pediatric transplants. Scientific publications were carefully assessed and, for each disease, the evidence for recommendation (from A to C) and the quality of the evidence (from 1 to 3) were defined. The recommendations include malignant and non-malignant hematological diseases, solid tumors, immunodeficiency, and storage diseases treated with hematopoietic stem cell transplants: either autologous or allogeneic from matched sibling donors or unrelated donors (adults or umbilical cord blood). Guidelines for reduced-intensity transplants, manipulated grafts or partially compatible donors were not included as there are no uniformly accepted recommendations. All indications are based on the best current knowledge which may change over time. Thus, this review should not be directly applied to patient care without taking into account the disease, donor and patient characteristics. Additionally, this paper should not be used as a document to limit patient access to transplant if correctly indicated. In this review we also point out differences between transplantation in adults and children and make some specific recommendations for pediatric transplants.

14698 downloads Cited 4 times in SciELO

11.

Transplante de células-tronco hematopoéticas em crianças e adolescentes com leucemias agudas
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Seber, Adriana

Revista Brasileira de Hematologia e Hemoterapia

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2010, Volume 32 N. 5 Pages 345 - 346

Abstract: Pt | Text: Pt | PDF: Pt

2589 downloads

12.

O transplante de células-tronco hematopoéticas na infância: situação atual e perspectivas
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Seber, Adriana

Revista Brasileira de Hematologia e Hemoterapia

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may 2009, Volume 31 Suppl. 1 Pages 59 - 67

Abstract: Pt En | Text: Pt En | PDF: Pt | PDF: En

O transplante de células-tronco hematopoéticas (TCTH) é uma opção terapêutica para um grande número de crianças com doenças malignas e não malignas. O objetivo deste artigo é apresentar a situação atual dos TCTH em pediatria para o tratamento de doenças hematológicas malignas, incluindo dados de nosso país e perspectivas futuras.

Hematopoietic stem cell transplantation is a treatment option for a large number of children with malignant and non-malignant diseases. The objective of this article is to present the current status of hematopoietic stem cell transplantation in the treatment of malignant hematological diseases in pediatrics, including results in Brazil, and future perspectives.

5295 downloads Cited 1 time in SciELO

13.

Terapia celular é a medicina do futuro?
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Ruiz, Milton A.

; Bydlowski, Sérgio P.

; Seber, Adriana

Revista Brasileira de Hematologia e Hemoterapia

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may 2009, Volume 31 Suppl. 1 Pages 1 - 1

Abstract: Pt | Text: Pt | PDF: Pt

2294 downloads

14.

Simplified flow cytometric assay to detect minimal residual disease in childhood with acute lymphoblastic leukemia
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Delbuono, Elizabete

; Maekawa, Yumi H.

; Latorre, Maria do Rosário D. O.

; Seber, Adriana

; Petrilli, Antonio Sergio

; Braga, Josefina A. P.

; Lee, Maria Lúcia M.

Revista Brasileira de Hematologia e Hemoterapia

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aug 2008, Volume 30 N. 4 Pages 281 - 286

Abstract: Pt En | Text: Pt En | PDF: Pt | PDF: En

A detecção de doença residual mínima (DRM) é um importante fator prognóstico na leucemia linfóide aguda (LLA) infantil e fornece informações sobre a resposta ao tratamento e o risco de recaída. Entretanto, os altos custos das técnicas utilizadas limitam seu uso nos países em desenvolvimento. Desta forma, realizamos um estudo prospectivo para avaliar a citometria de fluxo (CF), utilizando três fluorescências e um painel limitado de anticorpos monoclonais, como método de detecção de DRM em medula óssea (MO) e sangue periférico (SP) de crianças com LLA. Amostras de MO e SP de 40 crianças portadoras de LLA foram analisadas nos dias (d)14 e d28 da indução e nas semanas 24-30 da terapia de manutenção. Foram consideradas como DRM+ as amostras que apresentaram > 0,01% das células com o fenótipo aberrante (FA). Oitenta e sete por cento dos pacientes apresentaram FA ao diagnóstico. No d14, 56% das amostras de MO e 43% do SP apresentaram DRM. No d28, foi detectada DRM em 45% e 31% das amostras de MO e SP, respectivamente. A porcentagem de DRM na MO foi similar à do SP nos casos de LLA-T, mas aproximadamente dez vezes maior na LLA de precursor-B. Foi detectada DRM na MO de 44% e 39% dos pacientes que estavam remissão morfológica nos d14 e d28, respectivamente. Não foi demonstrada associação significante entre a presença de DRM e sexo, idade, leucometria inicial e linhagem celular. Esta técnica de detecção de DRM por CF é relativamente barata e pode ser aplicada em centros com recursos limitados.

The detection of minimal residual disease (MRD) is an important prognostic factor in childhood acute lymphoblastic leukemia (ALL) providing crucial information on the response to treatment and risk of relapse. However, the high cost of these techniques restricts their use in countries with limited resources. Thus, we prospectively studied the use of flow cytometry (FC) with a simplified 3-color assay and a limited antibody panel to detect MRD in the bone marrow (BM) and peripheral blood (PB) of children with ALL. BM and PB samples from 40 children with ALL were analyzed on days (d) 14 and 28 during induction and in weeks 24-30 of maintenance therapy. Detectable MRD was defined as > 0.01% cells expressing the aberrant immunophenotype as characterized at diagnosis among total events in the sample. A total of 87% of the patients had an aberrant immunophenotype at diagnosis. On d14, 56% of the BM and 43% of the PB samples had detectable MRD. On d28, this decreased to 45% and 31%, respectively. The percentage of cells with the aberrant phenotype was similar in both BM and PB in T-ALL but about 10 times higher in the BM of patients with B-cell-precursor ALL. Moreover, MRD was detected in the BM of patients in complete morphological remission (44% on d14 and 39% on d28). MRD was not significantly associated to gender, age, initial white blood cell count or cell lineage. This FC assay is feasible, affordable and readily applicable to detect MRD in centers with limited resources.

5396 downloads Cited 1 time in SciELO

15.

Síndrome mielodisplásica na infância
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Lopes, Luiz F.

; Lorand-Metze, Irene

; Mendes, Wellington L.

; Seber, Adriana

; Melo, Ligia N.

Revista Brasileira de Hematologia e Hemoterapia

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sep 2006, Volume 28 N. 3 Pages 226 - 237

Abstract: Pt En | Text: Pt En | PDF: Pt | PDF: En

O Grupo Cooperativo Brasileiro de Síndrome Mielodisplásica em Pediatria (GCB-SMD-PED) foi formado em janeiro de 1997 com o objetivo de estudar crianças (menores de 18 anos) com diagnóstico confirmado ou suspeita de mielodisplasia de todo o país. As SMD entretanto, por fazerem interfaces com as leucemias mielóides agudas - LMA), bem como com as doenças mieloproliferativas crônicas - DMPC), podem apresentar-se morfologicamente de várias formas, passíveis de confusão diagnóstica. Assim também, outras doenças com alteração hematológica podem trazer confusão e erros diagnósticos. Daí a necessidade da criação do GCB-SMD-PED para oferecer revisão e suporte no diagnóstico e nos exames complementares dos casos suspeitos de SMD na faixa pediátrica. Embora ainda se use a classificação FAB, duas novas classificações em pediatria foram recentemente propostas: a do Hospital for Sick Children, University of Toronto, Canadá, que propõe a "Classificação CCC" (categoria, citologia e citogenética), na qual foram utilizadas três características principais: categorias de origem "de novo", secundárias e/ou associadas a anormalidades constitucionais, critérios citológicos, com evidências ou não de displasia, e critérios citogenéticos, e a classificação proposta por Hasle e colaboradores, chamada de WHO pediátrica. Neste artigo serão apresentados os dados de 173 pacientes cadastrados no GCB-SMD-PED provenientes de 15 estados brasileiros (41 centros de tratamento em oncologia e hematologia pediátrica). De 1983 a 1997, 51 pacientes foram registrados de forma retrospectiva, e de janeiro de 1998 a fevereiro de 2003, 122 pacientes foram encaminhados ao grupo brasileiro e os seus dados coletados de forma prospectiva. Dos casos registrados e analisados, 93 tiveram confirmação de SMD. Em 36,5%, houve transformação para leucose aguda, sendo que a maioria sofreu transformação para LMA (82,3%) e menor porcentagem para LLA (17,7%). Quanto à evolução clínica dos pacientes encontramos: óbito em 54,8% dos casos, remissão espontânea em 5,4% e 16,1% encontrava-se em tratamento conservador. Infecções em pacientes com SMD e naqueles que sofreram transformação leucêmica foram as causas mais freqüentes de óbito (58,8%). Propomos também neste artigo uma abordagem de diagnóstico em genética e biologia molecular voltada para os casos infantis assim como uma revisão de literatura sobre transplante de medula óssea para os casos pediátricos, além de outros aspectos que diferem da abordagem feita para pacientes adultos.

The Brazilian Cooperative Study Group on Pediatric Myelodysplastic Syndromes (GCB-SMD-PED) started in January 1997 with the goal of studying under 18-year-old patients with MDS or suspected MDS from all over the country. Some primary or secondary disorders are incorrectly called MDS. Because of this the GCB-SMD-PED is a referral group in the country to review and also to give diagnostic support (morphology, genetics, etc.). Some groups still use the FAB classification but two new classifications for pediatric cases have been published: one from the Sick Children's Hospital, University of Toronto, Canada the "CCC Classification" (category, cytology and cytogenetic), and the WHO pediatric classification by Hasle et al. Our proposal here is to present data from the 173 pediatric cases which were referred to the GCB-SMD-PED from 15 states (41 centers). From 1983 to 1997, 51 pediatric cases were registered as retrospective cases and from January 1998 to February 2003, 122 prospective cases were registered. From these 173 cases, 93 where confirmed as MDS. In 36.5% of them there was a transformation into acute leukemia with 82.3% as AML and 17.7% as ALL. The follow up showed that 54.8% died, 5.4% had spontaneous remission and 16.1% were in treatment with no chemotherapy (just transfusion or conservative approach). Infections were the primary cause of death (58.8%). Additionally, in this article the diagnostic approach according to classical or molecular genetics is shown with a review of literature for bone marrow transplantation in pediatric cases and other aspects which are different from the approach offered to adult patients with MDS.

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