Design of the VIALE-M phase III trial of venetoclax and oral azacitidine maintenance therapy in acute myeloid leukemia.

Prevention of relapse is a major therapeutic challenge and an unmet need for patients with acute myeloid leukemia (AML). Venetoclax is a highly selective, potent, oral BCL-2 inhibitor that induces apoptosis in AML cells. When combined with azacitidine, it leads to prolonged overall survival and rapid, durable remissions in treatment-naive AML patients ineligible for intensive chemotherapy. VIALE-M is a randomized, double-blind, two-arm study to evaluate the safety and efficacy of venetoclax in combination with oral azacitidine (CC-486) as maintenance therapy in patients in complete remission with incomplete blood count recovery after intensive induction and consolidation therapies. The primary end point is relapse-free survival. Secondary outcomes include overall survival, minimal residual disease conversion and improvement in quality-of-life. Trial Registration Number: NCT04102020 (ClinicalTrials.gov).

Venetoclax combinations delay the time to deterioration of HRQoL in unfit patients with acute myeloid leukemia.

Phase 3 trials Viale-A and Viale-C evaluated health-related quality of life (HRQoL) in patients with AML unfit for intensive chemotherapy who received venetoclax (VEN) + (AZA) (Viale-A) or low-dose cytarabine (LDAC) (Viale-C) or placebo (PBO) + AZA or LDAC. Patient-reported outcomes included: EORTC QLQ-C30 global health status (GHS/QoL) and physical functioning (PF), PROMIS Cancer Fatigue Short Form 7a (Fatigue), and EQ-5D-5L health status visual analog scale (HS-VAS). Time to deterioration (TTD), defined as worsening from baseline in meaningful change thresholds (MCT) of ≥10, 5, or 7 points for GHS/QoL or PF, fatigue, and HS-VAS, respectively, was assessed; differences between groups were analyzed using Kaplan-Meier and unadjusted log-rank analyses. VEN + AZA vs PBO + AZA patients had longer TTD in GHS/QoL (P = 0.066) and fatigue (P = 0.189), and significantly longer TTD in PF (P = 0.028) and HS-VAS (P < 0.001). VEN + LDAC vs PBO + LDAC patients had significantly longer TTD in GHS/QoL (P = 0.011), PF (P = 0.020), and fatigue (P = 0.004), and a trend in HS-VAS (P = 0.057). Approximately 43%, 35%, 32%, and 18% of patients treated with VEN + AZA, AZA + PBO, VEN + LDAC, or LDAC + PBO, respectively, saw improvements >MCT in GHS/QoL. Overall, VEN may positively impact HRQoL in patients with AML ineligible for intensive chemotherapy, leading to longer preservation of functioning and overall health status.

6-month follow-up of VIALE-C demonstrates improved and durable efficacy in patients with untreated AML ineligible for intensive chemotherapy (141/150).

VIALE-C compared the safety and efficacy of venetoclax or placebo plus low-dose cytarabine (+LDAC) in patients with untreated AML ineligible for intensive chemotherapy. Overall, 211 patients were enrolled (n = 143, venetoclax; n = 68, placebo). At the primary analysis, the study did not meet its primary endpoint of a statistically significant improvement in overall survival (OS), however, ~60% of patients had been on study for ≤6-months. Here, we present an additional 6-months of follow-up of VIALE-C (median follow-up 17.5 months; range 0.1-23.5). Median OS was (venetoclax +LDAC vs. placebo +LDAC) 8.4 vs. 4.1 months (HR = 0.70, 95% CI 0.50,0.99; P = 0.040); a 30% reduction in the risk of death with venetoclax. Complete response (CR)/CR with incomplete hematologic recovery (CRi) rates were 48.3% vs. 13.2%. Transfusion independence rates (RBC) were 43% vs.19% and median event-free survival was 4.9 vs. 2.1 months (HR = 0.61; 95% CI 0.44,0.84; P = 0.002). These results represent improved efficacy over the primary analysis. Incidence of grade ≥3 adverse events were similar between study arms and overall safety profiles were comparable to the primary analysis. These data support venetoclax +LDAC as a frontline treatment option for patients with AML ineligible for intensive chemotherapy.This trial was registered at www.clinicaltrials.gov as #NCT03069352.

Venetoclax plus low-dose cytarabine in Japanese patients with untreated acute myeloid leukaemia ineligible for intensive chemotherapy.

BACKGROUND: In a multinational phase 3 trial (VIALE-C), venetoclax plus low-dose cytarabine prolonged overall survival vs placebo plus low-dose cytarabine in patients with newly diagnosed acute myeloid leukaemia ineligible for intensive chemotherapy, although it was not statistically significant. Herein, we assess the benefit of venetoclax plus low-dose cytarabine in the Japanese subgroup of VIALE-C patients (n = 27). METHODS: VIALE-C, a randomized (2:1), double-blind study (NCT03069352), enrolled untreated patients (≥18 years) with acute myeloid leukaemia. Patients received venetoclax (600 mg days 1-28, 4-day ramp-up in cycle 1) or placebo in 28-day cycles with low-dose cytarabine (20 mg/m2 days 1-10). The primary endpoint was median overall survival. RESULTS: In the Japanese subgroup, at a 6-month follow-up from the primary analysis, median overall survival for venetoclax (n = 18) and placebo (n = 9), plus low-dose cytarabine, was 4.7 and 8.1 months, respectively (hazard ratio, 0.928, 95% confidence intervals : 0.399, 2.156). The rate of complete remission plus complete remission with incomplete blood count recovery was higher with venetoclax plus low-dose cytarabine (44.4%) vs placebo plus low-dose cytarabine (11.1%). All patients experienced at least 1 adverse event. The most common grade ≥3 adverse events with venetoclax or placebo, plus low-dose cytarabine, were febrile neutropenia (50.0% vs 44.4%, respectively) and thrombocytopenia (27.8% vs 44.4%, respectively). Serious adverse events were reported in 50.0 and 33.3% of patients in the venetoclax and placebo, plus low-dose cytarabine arms, respectively; pneumonia was the most common (22.2% each). CONCLUSIONS: Limited survival benefit in the Japanese subgroup can be attributed to small patient numbers and to baseline imbalances observed between treatment arms, with more patients in the venetoclax plus low-dose cytarabine arm presenting poor prognostic factors. Venetoclax plus low-dose cytarabine was well tolerated in Japanese patients with acute myeloid leukaemia ineligible for intensive chemotherapy.

Venetoclax plus LDAC for newly diagnosed AML ineligible for intensive chemotherapy: a phase 3 randomized placebo-controlled trial.

Effective treatment options are limited for patients with acute myeloid leukemia (AML) who cannot tolerate intensive chemotherapy. Adults age ≥18 years with newly diagnosed AML ineligible for intensive chemotherapy were enrolled in this international phase 3 randomized double-blind placebo-controlled trial. Patients (N = 211) were randomized 2:1 to venetoclax (n = 143) or placebo (n = 68) in 28-day cycles, plus low-dose cytarabine (LDAC) on days 1 to 10. Primary end point was overall survival (OS); secondary end points included response rate, transfusion independence, and event-free survival. Median age was 76 years (range, 36-93 years), 38% had secondary AML, and 20% had received prior hypomethylating agent treatment. Planned primary analysis showed a 25% reduction in risk of death with venetoclax plus LDAC vs LDAC alone (hazard ratio [HR], 0.75; 95% confidence interval [CI], 0.52-1.07; P = .11), although not statistically significant; median OS was 7.2 vs 4.1 months, respectively. Unplanned analysis with additional 6-month follow-up demonstrated median OS of 8.4 months for the venetoclax arm (HR, 0.70; 95% CI, 0.50-0.98; P = .04). Complete remission (CR) plus CR with incomplete blood count recovery rates were 48% and 13% for venetoclax plus LDAC and LDAC alone, respectively. Key grade ≥3 adverse events (venetoclax vs LDAC alone) were febrile neutropenia (32% vs 29%), neutropenia (47% vs 16%), and thrombocytopenia (45% vs 37%). Venetoclax plus LDAC demonstrates clinically meaningful improvement in remission rate and OS vs LDAC alone, with a manageable safety profile. Results confirm venetoclax plus LDAC as an important frontline treatment for AML patients unfit for intensive chemotherapy. This trial was registered at www.clinicaltrials.gov as #NCT03069352.

Hantavirus pulmonary syndrome: prognostic factors for death in reported cases in Brazil.

Hantavirus pulmonary syndrome (HPS) was described for the first time in Brazil in 1993 and has occurred endemically throughout the country. This study analysed clinical and laboratory aspects as well as death-related factors for HPS cases in Brazil from 1993 to 2006. The investigation comprised a descriptive and exploratory study of the history of cases as well as an analytical retrospective cohort survey to identify prognostic factors for death due to HPS. A total of 855 Brazilian HPS cases were assessed. The majority of cases occurred during spring (33.5%) and winter (27.6%), mainly among young male adults working in rural areas. The global case fatality rate was 39.3%. The mean interval between the onset of symptoms and hospitalisation was 4 days and that between hospitalisation and death was 1 day. In the multiple regression analysis, adult respiratory distress syndrome and mechanical respiratory support were associated with risk of death; when these two variables were excluded from the model, dyspnoea and haemoconcentration were associated with a higher risk of death.

Linfoma de Hodgkin clássico na infância. Experiência de tratamento e fatores prognósticos / Classical Hodgkin?s lymphoma in childhood. Therapeutic experience and prognostic factors

Objetivos: Estimar as taxas de sobrevida global e livre de eventos em portadores de linfoma de Hodgkin (LH), bem como identificar fatores prognósticos. Métodos: Estudo de coorte retrospectivo, incluindo variáveis demográficas, laboratoriais, tipo histológico, estadiamento e tratamento de 107 pacientes menores de 18 anos de idade admitidos no Departamento de Pediatria do Centro de Tratamento e Pesquisa Hospital do Câncer, no período entre 1985 e 1995. Resultados: Dos pacientes, 81 (76%) eram do sexo masculino e 80% da raça branca. A média de idade foi 10 anos (2 a 18 anos). Adenomegalia cervical foi a principal queixa referida (68% dos pacientes) e 55% apresentavam tempo de queixa menor que seis meses. Os subtipos EN e CM foram encontrados em 43% e 41% dos casos, respectivamente. Os estádios clínicos II e III foram os mais frequentes (33% cada um). Os sítios metastáticos mais frequentes nos EC IV foram fígado (42%) e pulmão (38%). As taxas de SG e SLE em 10 anos foram de 82,4% e 82,5%, respectivamente. O estádio clínico se mostrou como fator prognóstico significativo para as SG e SLE. A análise univariada revelou a presença de sintomas B, nível de Hb £ 9,3 g/dl, leucócitos £ 6.100 mm3, plaquetas £ 274.000/mm3 e ocorrência de recaída como fatores de mau prognóstico, enquanto a análise múltipla mostrou como fatores prognósticos independentes a presença de sintomas B e contagem de plaquetas. Conclusões: A identificação de fatores prognósticos é valiosa para a adequada estratificação dos pacientes em grupos de risco, adequando-os a esquemas de tratamento que maximizem as taxas de cura e minimizem os efeitos colaterais tardios.

Hantaviruses and hantavirus pulmonary syndrome, Maranhao, Brazil.

To confirm circulation of Anajatuba virus in Maranhao, Brazil, we conducted a serologic survey (immunoglobulin G ELISA) and phylogenetic studies (nucleocapsid gene sequences) of hantaviruses from wild rodents and persons with hantavirus pulmonary syndrome. This virus is transmitted by Oligoryzomys fornesi rodents and is responsible for hantavirus pulmonary syndrome in this region.

[Clinical and laboratory profile of children living with vertically transmitted HIV/AIDS in a city in northeastern Brazily]. / Perfil clínico-laboratorial de crianças vivendo com HIV/AIDS por transmissão vertical em uma cidade do Nordeste brasileiro.

INTRODUCTION: Vertical transmission constitutes the main route for child infection by the HIV-1 virus (human immune deficiency virus). This study aimed to investigate the clinical and laboratory evolution of children with vertically transmitted HIV/AIDS. METHODS: This was a retrospective descriptive study based on data gathered from the medical records of all the children who were seen at a specialized care unit between January 1998 and June 2006. RESULTS: Eighty children who met the inclusion criteria were evaluated. In the cases 56 (70%) of the children, their mothers were diagnosed as HIV-positive after childbirth. The delivery was vaginal for 44 (55%) of the children. Fifty-six children (70%) were breastfed by their mothers for periods ranging from one to more than 12 months. Failure to use or incomplete use of the ACTG 076 protocol was documented in 63 (78.5%) of the cases. CONCLUSIONS: The findings from our study are a cause for considerable concern and show failures of medical care for mothers and children, particularly with regard to prevention of transmission.

Perfil clínico-laboratorial de crianças vivendo com HIV/AIDS por transmissão vertical em uma cidade do Nordeste brasileiro / Clinical and laboratory profile of children living with vertically transmitted HIV/AIDS in a city in northeastern Brazily

INTRODUÇÃO: a transmissão vertical constitui a principal via de infecção infantil pelo vírus HIV-1 (vírus da imunodeficiência humana). A presente pesquisa tem como objetivo estudar a evolução clínica e laboratorial de crianças vivendo com HIV/AIDS decorrente da transmissão vertical. MÉTODOS: trata-se de um estudo descritivo, retrospectivo, realizado a partir da coleta de dados em prontuário médico de todas as crianças atendidas em um Serviço de Assistência Especializada, no período de janeiro de 1998 a junho de 2006. RESULTADOS: foram avaliadas 80 crianças que preencheram critérios de inclusão. Observou-se que em 56 (70 por cento) crianças, o diagnóstico da infecção pelo HIV na mãe deu-se após o parto e que em 44 (55 por cento) o parto foi via vaginal. Amamentação ao seio materno foi documentada em 56 (70 por cento) crianças e esta variou de um mês até mais de 12 meses. A não utilização ou uso incompleto do Protocolo ACTG 076 foi documentado em 63 (78,5 por cento) casos. CONCLUSÕES: os dados observados em nosso estudo são bastante preocupantes e revelam falha na assistência materno-infantil, especialmente voltada para prevenção da transmissão.

INTRODUCTION: Vertical transmission constitutes the main route for child infection by the HIV-1 virus (human immune deficiency virus). This study aimed to investigate the clinical and laboratory evolution of children with vertically transmitted HIV/AIDS. METHODS: This was a retrospective descriptive study based on data gathered from the medical records of all the children who were seen at a specialized care unit between January 1998 and June 2006. RESULTS: Eighty children who met the inclusion criteria were evaluated. In the cases 56 (70 percent) of the children, their mothers were diagnosed as HIV-positive after childbirth. The delivery was vaginal for 44 (55 percent) of the children. Fifty-six children (70 percent) were breastfed by their mothers for periods ranging from one to more than 12 months. Failure to use or incomplete use of the ACTG 076 protocol was documented in 63 (78.5 percent) of the cases. CONCLUSIONS: The findings from our study are a cause for considerable concern and show failures of medical care for mothers and children, particularly with regard to prevention of transmission.

An outbreak of bat-transmitted human rabies in a village in the Brazilian Amazon.

During 45 days without electrical power, 57 individuals (8.7% of the population) from the village of Antônio Dino (municipality of Turiaçu, Northeastern Brazil) were attacked by bats and 16 died from human rabies. The aim of the study was to analyze the factors associated with bat attacks and the development of human rabies. Of the 46 individuals, who suffered bat attacks, 36 (78.3%) were under 17 years of age. The risk factors associated with bat attacks were age under 17 years, having observed bats inside the bedroom and having been without electrical power in the house. Age under 17 years and having been without electrical power in the house were factors associated with human rabies.

An outbreak of bat-transmitted human rabies in a village in the Brazilian Amazon / Surto de raiva humana transmitida por morcegos em povoado da Amazônia brasileira

During 45 days without electrical power, 57 individuals (8.7 percent of the population) from the village of Antônio Dino (municipality of Turiaçu, Northeastern Brazil) were attacked by bats and 16 died from human rabies. The aim of the study was to analyze the factors associated with bat attacks and the development of human rabies. Of the 46 individuals, who suffered bat attacks, 36 (78.3 percent) were under 17 years of age. The risk factors associated with bat attacks were age under 17 years, having observed bats inside the bedroom and having been without electrical power in the house. Age under 17 years and having been without electrical power in the house were factors associated with human rabies.

Durante 45 dias sem energia elétrica, 57 indivíduos (8,3 por cento da população) da localidade Antônio Dino, município de Turiaçu, MA, foram atacados por morcegos e 16 morreram de raiva humana. O objetivo deste estudo foi analisar os fatores associados aos ataques por morcegos e ao desenvolvimento de raiva humana. Dos 46 indivíduos que sofreram ataque por morcegos, 36 (78,3 por cento) tinham menos de 17 anos de idade. Os fatores de risco associados a ataques por morcegos foram idade inferior a 17 anos, ter observado morcego dentro do quarto e haver ficado sem energia elétrica no domicílio. Idade inferior a 17 anos e ter ficado sem energia elétrica no domicílio foram fatores associados à raiva humana.

Influência das características clínicas e biológicas no prognóstico de lactentes com leucemia linfocítica aguda: proposta de um escore preditivo com inclusão de polimorfismo genético / Clinical and biologic characteristics on the prognosis of infants with acute lymphoblastic leukemia. proposal of a predictive score including genetic polymorphism

Leucemias agudas da infância formam um grupo heterogêneo de doenças hematológicas malignas. Revisando-se sua epidemiologia descritiva e analítica, pode-se enfatizar que além de fatores clínicos e laboratoriais, aspectos biológicos, como subtipos morfológicos, achados citogenéticos e marcadores moleculares, também interferem de maneira significativa na sobrevida dos pacientes. De forma geral, leucemias em lactentes têm características epidemiológicas, biológicas e clínicas distintas em relação aos pacientes mais velhos... Objetivos: Analisar as características clínicas e biológicas de pacientes lactentes (< 24 meses de vida), identificar fatores prognósticos clínicos e biológicos, construir escore prognóstico e estimar a probabilidade de sobrevida global (SG) para o grupo estudado. Casuística e métodos: Retrospectivamente, foram analisadas as informações clínicas e biológicas de 249 pacientes com leucemia linfóide aguda (LLA), menores de 24 meses de idade e inscritos na Divisão de Medicina Experimental, Centro de Pesquisa (MedEx, CPq) do Instituto Nacional de Câncer, Rio de Janeiro, entre janeiro de 1990 e dezembro de 2003. Resultados: O grupo apresentou as seguintes características: discreta predominância do sexo feminino; 74% de raça branca; mediana de idade de 14,1 meses (57% eram lactentes ≤ 14 meses de idade); para pacientes ≤ 14 meses, 57% tinham entre 6 e 14 meses e 26% tinham ≤ 3 meses; 55% dos pacientes apresentavam contagem leucocitária menor que 50x109/l; 54% apresentavam CD10 negativo; 50% dos casos tinham imunofenótipo B comum e 42% eram pró-B ... Conclusões: Na casuística estudada, os resultados obtidos mostraram que a LLA em pacientes lactentes origina-se de células mais imaturas e preditivas de doença mais agressiva. A continuação do estudo das características biológicas de forma contínua e ativa se faz necessária em nosso país, assim como o estabelecimento de tratamentos padronizados e feitos cooperativamente.

Childhood acute leukemias are a heterogeneous group of malignant hematologic diseases. Reviewing the descriptive and analytical epidemiology of them, it can be observed that besides clinical and laboratory factors, biological aspects, such as morphological subtypes, cytogenetic features, and molecular markers, interfere significantly on the survival of patients. In a general view, leukemia in infants has different epidemiological, biological, and clinical characteristics compared to older patients. The majority of cases present with high tumor load at diagnosis (high white blood cell count on peripheral blood, hepatosplenomegaly, and CNS involvement), with leukemic cells being phenotypically early B-cell precursor (CD34+, CD19+), lacking the CD10 antigen, while often expressing myeloid precursor antigens (CD15/CD65s) and increased myeloperoxidase expression. It is known that the combination of factors presented at diagnosis allows the identification of different risk groups, defining each patient prognosis and consequently determining the best therapeutic approach. Objectives: To analyze clinical and biological characteristics in infants (< 24 month year old) in order to identify clinical and biological prognostic factors, to prepare a prognostic score, and to estimate overall survival (OS) to the cohort. Patients and methods: Two hundred and forty-nine acute lymphoblastic leukemia (ALL) patients were retrospectively analyzed according to their clinical and biological information. They were younger than 24 months and enrolled at the Divisão de Medicina Experimental, Centro de Pesquisa (MedEx, CPq) of Instituto Nacional de Câncer, Rio de Janeiro, from January 1990 to December 2003. Results: The following characteristics were found: small predominance of females; 74% of Caucasian with median age of 14.1 months (57% younger than 14 months); among patients younger than 14 months, 57% were 6 to 14 months old and 26% were younger than 3 months old; 55% presented white blood count < 50x109/l, negative CD10; 50% common B immunophenotype and 42% early B immunophenotype according to the EGIL classification; MTHFRC677T or MTHFRT677T genotypes were more frequent than MTHFRC677C; MTHFRA1298A was more frequent than genotypes involving C allele and 81% of patients received treatment according to GBTLI protocols. Patients younger than 14 months old had statistically significant association with antigen CD10, EGIL early B-cell classification, and presence of MLL gene rearrangements. MLL rearrangements presented significantly association with negative CD10 and EGIL early B-cell classification; 93% of EGIL early B-cell classification patients presented negative CD10. The entire group presented 45% 5-year overall survival and the worst survival rates were observed in patients younger than 14 months, patients with white blood count > 100x109/l at diagnosis, negative CD10, those with EGIL early B-cell classification, and patients who were treated with a protocol different from GBTLI. When only patients younger than 14 months were analyzed, the OS was worse in those with white blood count > 100x109/l, negative CD10, patients with EGIL early B-cell classification, and those who presented with MTHFRT677T or MTHFRC677T genotype. For patients under 14 months, the prognostic score model identified the following characteristics as factors that can influence overall survival statistically significant: high white blood cells, negative CD10, pro-B EGIL immunophenotype and MTHFRC677T or MTHFRT677T genotype. Conclusions: Results have shown that on this cohort ALL originates from very immature cells and can predict an aggressive disease. It is imperative to continue studying its biological characteristics in our country as well as to establish standardized and co-operative treatments.

Histiocitose de células de Langerhans com envolvimento ungueal em criança: relato de caso / Langerhans cell histiocytosis with nail involvement in childhood: a case report

A histiocitose de células de Langerhans pode apresentar-se de diversas formas clínicas. Neste trabalho, os autores relatam caso de menino de três anos de idade com queixa de otite média crônca e tumoração na região da mastóide direita. O exame anatomopatológico revelou histiocitose. O paciente apresentava dermatite importante no couro cabeludo e alterações distróficas com onicólise, pústulas e deformidades ungueais nos dedos das mãos e pés. As lesões responderam à terapia antineoplásica. O aparecimento de lesões distróficas ungueais na histiocitose de células de Langerhans é raro em crianças. Esse caso clínico sugere que o tratamento com terapia antineoplásica pode ser eficaz.

Langerhans cell histiocytosis may appear in a variety of ways. The authors present the case report of a 3-year-old white boy with a main complaint about chronic media otitis and a tumor lesion in right mastoid bone. Pathology revealed histiocytosis. The patient had severe dermatitis on the scalp and dystrophic changes with onycholysis, pustules, and nail plate deformity underneath all fingernails and toenails. These lesions responded to antineoplastic therapy. Development of nail dystrophics in Langerhans cell histiocytosis is unusual in children. This case suggests that treatment with antineoplastic therapy might be effective.

Bilateral renal metastases from osteosarcoma: A case report and review of the literature.

Improvements in multimodal therapy for osteosarcoma (OS) have increased event-free and overall survival. But have also led to a greater number of recurrences in uncommon sites. We report a young adult with OS who developed late bilateral renal relapse. Late recurrences to the kidneys have a more aggressive clinical behavior and poor prognosis documented by 15 cases of OS metastastic to the kidney in the literature. Two of those patients had a long survival after chemotherapy and surgery. This suggests that the disease can be controlled with early detection and treatment.

Síndrome mielodisplásica na infância / Myelodysplastic syndromes in childhood

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 por cento, houve transformação para leucose aguda, sendo que a maioria sofreu transformação para LMA (82,3 por cento) e menor porcentagem para LLA (17,7 por cento). Quanto à evolução...

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 percent of them there was a transformation into acute leukemia with 82.3 percent as AML and 17.7 percent as ALL. The follow up showed that 54.8 percent died, 5.4 percent had spontaneous remission and 16.1 percent were in treatment with no chemotherapy (just transfusion or conservative approach). Infections were the primary cause of death (58.8 percent). 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.