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2.
Blood Cancer J ; 4: e252, 2014 Oct 17.
Artigo em Inglês | MEDLINE | ID: mdl-25325302

RESUMO

The superiority of the pediatric protocol for adolescents with acute lymphoblastic leukemia (ALL) has already been demonstrated, however, its efficacy in young adults remains unclear. The ALL202-U protocol was conducted to examine the efficacy and feasibility of a pediatric protocol in adolescents and young adults (AYAs) with BCR-ABL-negative ALL. Patients aged 15-24 years (n=139) were treated with the same protocol used for pediatric B-ALL. The primary objective of this study was to assess the disease-free survival (DFS) rate and its secondary aims were to assess toxicity, the complete remission (CR) rate and the overall survival (OS) rate. The CR rate was 94%. The 5-year DFS and OS rates were 67% (95% confidence interval (CI) 58-75%) and 73% (95% CI 64-80%), respectively. Severe adverse events were observed at a frequency that was similar to or lower than that in children treated with the same protocol. Only insufficient maintenance therapy significantly worsened the DFS (hazard ratio 5.60, P<0.001). These results indicate that this protocol may be a feasible and highly effective treatment for AYA with BCR-ABL-negative ALL.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Adolescente , Adulto , Fatores Etários , Protocolos de Quimioterapia Combinada Antineoplásica/antagonistas & inibidores , Intervalo Livre de Doença , Feminino , Humanos , Japão , Masculino , Estudos Prospectivos , Indução de Remissão , Taxa de Sobrevida , Adulto Jovem
3.
Haemophilia ; 19(3): 378-84, 2013 May.
Artigo em Inglês | MEDLINE | ID: mdl-23379934

RESUMO

Severe heritable protein C (PC) deficiency is quite rare, although heterozygous PROC mutation is the second leading cause of genetic predisposition to thrombosis in Japanese adults. The aim of the study was to search the optimal management, the paediatric onset and outcomes of PC deficiency were characterized in Japan. The genetic study, postmarketing survey of activated PC(aPC) concentrate (Anact(®)C) and intensive review in Japan for 20 years enabled the analysis of the disease onset, genotype, treatment and prognosis. Symptomatic PC deficiency was determined in 27 Japanese children. All but two patients presented within 16 days after birth (three prenatal and six neonatal onsets). Postnatal-onset cases had normal growth at full-term delivery. Of the 27 patients, 19 suffered intracranial thrombosis or haemorrhage (ICTH) (three foetal hydrocephalies), 16 developed purpura fulminans (PF) and 10 had both at the first presentation. ICTH preceded PF in both affected cases. Low PC activities of 18 mothers and/or 12 fathers indicated 20 inherited PC deficiencies (2 homozygotes, 11 compound heterozygotes and 7 heterozygotes) and seven unidentified causes of PC deficiency. Nine of 11 patients studied had PROC mutations. Four unrelated patients (50%) carried PC nagoya (1362delG). No PC-deficient parents had experienced thromboembolism. Of the 18 patients with aPC therapy, two died and eight evaluable survivors had neurological sequelae. This first comprehensive study of paediatric PC deficiency suggested that perinatal ICTH was the major presentation, occurring earlier than neonatal PF. PC nagoya was prevalent in paediatric, but not adult, patients in Japan. Early maternal screening and optimal PC therapy are required for newborns at risk of PC deficiency.


Assuntos
Deficiência de Proteína C/tratamento farmacológico , Proteína C/uso terapêutico , Adolescente , Anticoagulantes/uso terapêutico , Criança , Pré-Escolar , Feminino , Genótipo , Heterozigoto , Homozigoto , Humanos , Lactente , Recém-Nascido , Japão , Masculino , Proteína C/genética , Deficiência de Proteína C/genética , Deficiência de Proteína C/patologia , Púrpura Fulminante/tratamento farmacológico , Púrpura Fulminante/patologia , Trombose/tratamento farmacológico , Trombose/patologia , Resultado do Tratamento , Tromboembolia Venosa/tratamento farmacológico , Tromboembolia Venosa/patologia
4.
Neuropediatrics ; 38(6): 317-9, 2007 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-18461510

RESUMO

A 1-year and 11-month-old female infant with bilateral lesions of the thalamus, basal ganglia, cerebellar and brainstem disease died from heart failure 9 days after being administered a measles vaccination. She had a high fever, hypocarnitinemic and non-ketotic hypoglycemia, serum levels of total carnitine 7.4 micromol/L, free carnitine 5.6 micromol/L, acylcarnitine 1.8 micromol/L and glucose 13 mg/dL. Due to feeding difficulty, the patient, however, had been administered parenteral elementary nutrition through a feeding tube since early infancy. The commercially available parenteral nutrition solutions do not contain carnitine. A secondary carnitine deficiency followed by non-ketotic hypoglycemia-related heart failure may readily develop even in a patient without valproic acid, during high fever.


Assuntos
Carnitina/deficiência , Insuficiência Cardíaca/etiologia , Hipoglicemia/etiologia , Vacina contra Sarampo/efeitos adversos , Nutrição Parenteral , Convulsões Febris/etiologia , Feminino , Humanos , Lactente , Imageamento por Ressonância Magnética
5.
Bone Marrow Transplant ; 27(7): 767-9, 2001 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-11360120

RESUMO

We describe here the case of an 8-year-old girl with Fanconi anemia (FA) whose hematopoiesis was successfully restored by unrelated umbilical cord blood (UCB) transplantation. The patient became resistant to androgen therapy, and developed intracranial hemorrhage and dyserythropoiesis. Her hematopoietic recovery after the transplantation was excellent and a complete chimerism has been durably maintained. UCB should be considered as a stem cell source for transplantation when a patient with FA does not have an HLA-identical unaffected sibling donor.


Assuntos
Anemia de Fanconi/terapia , Hematopoese , Transplante de Células-Tronco Hematopoéticas , Pré-Escolar , Intervalo Livre de Doença , Anemia de Fanconi/complicações , Feminino , Sangue Fetal , Sobrevivência de Enxerto , Histocompatibilidade , Humanos , Hemorragias Intracranianas/etiologia , Quimeras de Transplante
6.
Cell ; 102(2): 199-209, 2000 Jul 21.
Artigo em Inglês | MEDLINE | ID: mdl-10943840

RESUMO

Angiogenesis is an important event for embryonic organogenesis as well as for tissue repair in the adult. Here, we show that hematopoietic stem cells (HSCs) play important roles for angiogenesis during embryogenesis. To investigate the role of HSCs in endothelial cell (EC) development, we analyzed AML1-deficient embryos, which lack definitive hematopoiesis. These embryos showed defective angiogenesis in the head and pericardium. Para-aortic splanchnopleural (P-Sp) explant cultures on stromal cells (P-Sp culture) did not generate definitive hematopoietic cells and showed defective angiogenesis in the AML1 null embryo. Disrupted angiogenesis in P-Sp cultures from AML1 null embryos was rescued by addition of HSCs or angiopoietin-1 (Ang1). HSCs, which express Ang1, directly promoted migration of ECs in vivo and in vitro. These results indicate that HSCs are critical for angiogenesis.


Assuntos
Células-Tronco Hematopoéticas/fisiologia , Neovascularização Fisiológica/fisiologia , Proteínas Proto-Oncogênicas , Angiopoietina-1 , Animais , Linhagem Celular , Subunidade alfa 2 de Fator de Ligação ao Core , Técnicas de Cultura , Proteínas de Ligação a DNA/genética , Expressão Gênica , Hematopoese , Glicoproteínas de Membrana/genética , Glicoproteínas de Membrana/fisiologia , Camundongos , Camundongos Endogâmicos C57BL , Camundongos Knockout , Camundongos Mutantes , Proteínas de Neoplasias/metabolismo , Pericitos/metabolismo , Molécula-1 de Adesão Celular Endotelial a Plaquetas/metabolismo , Receptores Proteína Tirosina Quinases/metabolismo , Receptores de Fatores de Crescimento/metabolismo , Receptores de Fatores de Crescimento do Endotélio Vascular , Fatores de Transcrição/genética
7.
Brain Dev ; 22(3): 196-8, 2000 May.
Artigo em Inglês | MEDLINE | ID: mdl-10814905

RESUMO

To elucidate a relationship between neuronal anaplasia, tumor proliferation, and ganglioside contents, we quantified gangliosides by HPTLC in tumors of neuroepithelial tissues at different grade, i. e. peripheral primitive neuroectodermal tumor (PPNET, grade IV), ependymoma (EPEN, grade III), neuroblastoma (NB, grade IV), and dysembryoplastic neuroepithelial tumor (DNT, grade I). PPNET, the most undifferentiated tumor examined had lowest concentration of total lipid-bound sialic acid. GM3 was the major ganglioside in all undifferentiated tumors (46-72.7% of total lipid-bound sialic acid). GD3 was an another component in PPNET and EPEN (7.2-17.3%). Concentration of a complex gangliosides GM1 was decreased in all tumor tissues and those of GT1a, GD1b and GT1b were decreased in tumors of high grade. The results suggest that the composition of gangliosides could be of considerable value in refining the classification of neuroepithelial tumors in infancy and childhood.


Assuntos
Biomarcadores Tumorais/análise , Biomarcadores Tumorais/metabolismo , Neoplasias Encefálicas/fisiopatologia , Gangliosídeos/análise , Gangliosídeos/metabolismo , Tumores Neuroectodérmicos Primitivos/fisiopatologia , Adolescente , Encéfalo/embriologia , Encéfalo/patologia , Encéfalo/fisiopatologia , Neoplasias Encefálicas/metabolismo , Neoplasias Encefálicas/patologia , Criança , Pré-Escolar , Ependimoma/metabolismo , Ependimoma/patologia , Ependimoma/fisiopatologia , Feminino , Humanos , Lactente , Masculino , Neuroblastoma/metabolismo , Neuroblastoma/patologia , Neuroblastoma/fisiopatologia , Tumores Neuroectodérmicos Primitivos/metabolismo , Tumores Neuroectodérmicos Primitivos/patologia , Tumores Neuroectodérmicos Primitivos Periféricos/metabolismo , Tumores Neuroectodérmicos Primitivos Periféricos/patologia , Tumores Neuroectodérmicos Primitivos Periféricos/fisiopatologia , Neurônios/química , Neurônios/metabolismo , Neurônios/patologia
8.
J Pediatr Surg ; 33(10): 1571-3, 1998 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-9802819

RESUMO

Wandering spleen is an uncommon condition, showing a splenic hypermobility that results from laxity or maldevelopment of its suspensory ligament. This condition results in constant danger of splenic torsion and infarction. Wandering spleen was diagnosed in a 2-year-old girl who complained of intermittent abdominal pain, and viability of the spleen was confirmed by doppler ultrasound scan. Elective laparoscopic splenopexy was performed by anchoring the spleen wrapped in absorbable mesh. To our knowledge, this is the first reported case of laparoscopic splenopexy for wandering spleen. The authors believe that this approach is a safe and effective procedure for wandering spleen with the advantage of minimally invasive surgery.


Assuntos
Laparoscopia , Baço/cirurgia , Esplenopatias/cirurgia , Pré-Escolar , Feminino , Humanos , Radiografia , Baço/diagnóstico por imagem , Esplenopatias/diagnóstico por imagem
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