No abstract

Studies have shown that autologous hematopoietic SCT (HSCT) can be used as an intensive immunosuppressive therapy to treat refractory patients and to prevent the progression of multiple sclerosis (MS). This is a prospective multicentric Brazilian MS trial comparing two conditioning regimens: BEAM/horse ATG and CY/ rabbit ATG. Most (80.4%) of the 41 subjects in the study had the secondary progressive MS subtype and the mean age was 42 years. The baseline EDSS score in 58.5% of the subjects was 6.5 and 78% had a score of 6.0 or higher, respectively. The complication rate during the intratransplantation period was 56% for all patients: 71.4% of the patients in the BEAM/hATG group and 40% in the CY/rATG group (P ¼ 0.04). Three subjects (7.5%) died of cardiac toxicity, sepsis and alveolar hemorrhage, all of them in the BEAM/ATG group. EFS was 58.54% for a ll patients: 47% in the BEAM/hATG group and 70% in the CY/rATG group (P ¼ 0.288). In conclusion, the CY/rATG regimen seems to be associated with similar outcome results, but presented less toxicity when compared with the BEAM/hATG regimen. Long-term followup would be required to fully assess the differences in therapeutic effectiveness between the two regimens.

No abstract

Music therapy proved to be a strong ally in the treatment of patients undergoing allo-HSCT, providing bio-psychosocial welfare.

The authors retrospectively assess the autopsy findings of central nervous system (CNS) infections in marrow transplant recipients. From July 1987 to June 1998, 845 patients at our institution were submitted to bone marrow transplantation (BMT). The CNS of 180 patients was studied through autopsy and these patients had their medical records reviewed. Twenty-seven (15%) patients presented brain parenchyma infection. Fungi were isolated in approximately 60% of the cases. Mean survival time was 153 days (0-1,264 days) and the majority of the patients died during the first 3 months after BMT (18 cases; 67%). Aspergillus sp. were the most prevalent fungi (approximately 30%), followed by Candida sp. infection (approximately 18%). There was one case of Fusarium sp. infection and two cases of unidentified fungus. All patients with fungal infections had documented involvement at widespread sites. Toxoplasma gondii encephalitis was demonstrated in 8 patents (approximately 30%). Bacterial abscesses were responsible for approximately 11% of the findings. Eleven (41%) of the 27 patients died secondary to cerebral causes. These results show that infectious compromise of the CNS following BMT is a highly fatal event, caused mainly by fungi and T. gondii. Furthermore, they provide a likely guide to the possible causes of brain abscesses following BMT.

The Brazilian Ministry of Health created the National Neonatal Screening Program under ruling no. 822/2001, including

-Bone marrow transplantation (BMT) is a therapeutic option for patients with genetic storage diseases. Between 1979 and 2002, eight patients, four females and four males (1 to 13 years old) were submitted to this pro c e d u re in our center. Six patients had mucopolysaccharidosis (MPS I in 3; MPS III in one and MPS VI in 2), one had adre n o l e u k o d y s t rophy (ALD) and one had Gaucher disease. Five patients had related and three unrelated BMT donor. Three patients developed graft versus host disease (two MPS I and one MPS VI) and died between 37 and 151 days after transplantation. Five patients survived 4 to 16 years after transplantation. Three patients improved (one MPS I; one MPS VI and the Gaucher disease patient), one patient had no disease progression (ALD) and in one patient this procedure did not change the natural course of the disease (MPS III).KEY WORDS: storage diseases, bone marrow transplantation, genetic neurological diseases, mucopolysaccharidosis, adrenoleukodystrophy, Gaucher disease.Transplante de medula óssea em pacientes com doença de acúmulo: experiência de um país em desenvolvimento RESUMO -O transplante de medula óssea é uma opção terapêutica para os pacientes com doenças de acú-mulo. Entre 1979 e 2002, oito pacientes, quatro femininos e quatro masculinos (entre um e 13 anos de idade) foram submetidos a este procedimento em nosso centro. Seis pacientes apresentavam mucopolissacaridose (MPS I em 3; MPS III em um e MPS VI em 2), um paciente apresentava adre n o l e u c o d i s t rofia e um apresentava doença de Gaucher. Cinco pacientes receberam o transplante de doador aparentado e três de doador não aparentado. Três pacientes desenvolveram doença do enxerto versus hospedeiro (dois com MPS I e um com MPS VI) e faleceram entre 37 e 151 dias após o transplante. Cinco pacientes sobreviveram entre 4 e 16 anos após o transplante. Três tiveram melhora clínica (um MPS I, um MPS VI e o paciente com doença de Gaucher), um paciente não apresentou pro g ressão da doença (adre n o l e u c o d i s t rofi a) e um paciente não teve alteração da história natural da doença (MPS III).

Anemia Fanconi (AF) é uma síndrome autossômica recessiva, caracterizada por pancitopenia progressiva com hipoplasia de MO, em associação com várias anormalidades constitucionais, tendo como único recurso terapêutico com possibilidade potencial de cura o transplante de medula óssea, e sendo tais pacientes propensos ao desenvolvimento de malignidades hematológicas e carcinoma de células escamosas (CEC) em diversos locais: reto, vagina, cérvice, esôfago, cavidade bucal, faringe ou pele, mas especialmente em cabeça e pescoço. Relatamos aqui três casos de pacientes portadores de AF, que após TMO desenvolveram CEC em língua. Além disso, mencionamos fatores de risco relatados para tal evento, como diagnóstico de AF, condicionamento pré-transplante (quimioterápicos e irradiação), terapia com drogas imunossupressoras para tratamento de doença enxerto contra hospedeiro (DECH) aguda ou crônica, sexo e idade avançada. Além do que, discorremos sobre a existência de três mecanismos postulados que predispõem indivíduos com AF ao desenvolvimento de neoplasia: (1) defeito na reparação do DNA; (2) defeito na detoxificação de radicais de oxigênio; e (3) imunodeficiência. Rev. bras. hematol.