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1.
Hamostaseologie ; 34 Suppl 1: S5-8, 2014.
Artigo em Inglês | MEDLINE | ID: mdl-25382771

RESUMO

Immune tolerance induction (ITI) in patients with haemophilia B and inhibitors may be complicated by anaphylactic reactions and nephrotic syndrome with lower success rates than in haemophilia A (25% vs. 50-90%). According to case reports, immunosuppressive therapy in addition to high doses of factor IX (FIX) appears to be promising. We report an 18-year-old patient with severe haemophilia B and a FIX inhibitor with a maximum titre of 2.6 Bethesda units and allergic skin reactions to FIX infusions. At 5 years of age, this patient already had a FIX inhibitor with allergic reactions to FIX and activated prothrombin complex concentrate. ITI at 11 years of age with high-dose FIX, dexamethasone, rituximab, mycophenolate mofetil and intravenous immunoglobulins had induced a sustained response until the current presentation. The patient was restarted on the same ITI regimen with aforementioned immunosuppressants, which were initiated one week before high-dose FIX. No allergic reactions, nephrotic syndrome or serious infection occurred during ITI. The FIX inhibitor was undetectable after five weeks of treatment and remained so until 19 months of follow-up.


Assuntos
Inibidores dos Fatores de Coagulação Sanguínea/sangue , Fator IX/administração & dosagem , Hemofilia B/tratamento farmacológico , Hemofilia B/imunologia , Tolerância Imunológica/imunologia , Imunossupressores/administração & dosagem , Adolescente , Inibidores dos Fatores de Coagulação Sanguínea/imunologia , Quimioterapia Combinada , Fator IX/imunologia , Feminino , Humanos , Tolerância Imunológica/efeitos dos fármacos , Imunossupressores/imunologia , Recidiva , Resultado do Tratamento
2.
Klin Padiatr ; 222(3): 175-9, 2010 May.
Artigo em Inglês | MEDLINE | ID: mdl-20514623

RESUMO

BACKGROUND: Germ cell tumors (GCT) situated in the head and neck region are very rare and occur predominantely in newborns or young infants. Recurrent CTs are often resectable only by mutilating surgery and the need for alternative treatment strategies is obvious. In this situation radiation therapy is the most important treatment option for loco-regional tumor control, but bear in this area the risk of possible impairment of brain function and face deformation as long term effects. CASE REPORT: In a girl with a connatal expansive growing teratoma of the skull the tumor recurred in spite of repeated surgery as mixed malignant GCT at the age of 15 months. Tumor control could not be achieved with chemotherapy and additional surgery seemed not promising. Therefore high dose proton beam therapy (PT) (54 Gy) has been administered to the child at the age of 22 months and led to local tumor control with only mild side effects. CONCLUSION: PT treatment may be an option for specific clinical conditions in germ cell tumors where local tumor control cannot be achieved by chemotherapy and/or surgery and long lasting side effects of conventional radiotherapy due to tumor localization and age have to be considered. However, PT should be implemented in treatment protocols for specific situations to guarantee supervised application, central documentation and follow-up.


Assuntos
Recidiva Local de Neoplasia/radioterapia , Neoplasias Orbitárias/congênito , Neoplasias Orbitárias/radioterapia , Terapia com Prótons , Planejamento da Radioterapia Assistida por Computador , Neoplasias da Base do Crânio/congênito , Neoplasias da Base do Crânio/radioterapia , Teratoma/congênito , Teratoma/radioterapia , Blefaroptose/etiologia , Pré-Escolar , Craniotomia , Feminino , Seguimentos , Humanos , Lactente , Imageamento por Ressonância Magnética , Invasividade Neoplásica , Recidiva Local de Neoplasia/diagnóstico , Recidiva Local de Neoplasia/cirurgia , Neoplasias Orbitárias/diagnóstico , Neoplasias Orbitárias/cirurgia , Lesões por Radiação/etiologia , Dosagem Radioterapêutica , Radioterapia Adjuvante , Reoperação , Neoplasias da Base do Crânio/diagnóstico , Neoplasias da Base do Crânio/cirurgia , Teratoma/diagnóstico , Teratoma/cirurgia
3.
Hamostaseologie ; 29(2): 143-8, 2009 May.
Artigo em Inglês | MEDLINE | ID: mdl-19404524

RESUMO

UNLABELLED: We have prospectively evaluated the biologic response to desmopressin (DDAVP) in 28 children with type 2 von Willebrand disease (VWD) in correlation with the phenotype and the molecular defect of VWF. The diagnosis of VWD type 2 was mainly based on VWF functional parameters and/or an aberrant VWF multimer pattern. Seventeen different mutations were identified (6 of them novel). No response with respect to the functional parameters VWF:RCo and/or VWF:CB was seen in patients with severe abnormality of the VWF multimer pattern. One patient with VWD type 2A phenotype IIC Miami did not respond with respect to VWF:CB, but showed a good response of VWF:Ag and FVIII:C as expected. Interestingly he showed a persistently high level of VWF:Ag and FVIII:C up to 4 hours after DDAVP infusion. Patients with minor alterations of multimer structure and particular mutations responded well to DDAVP, whereas patients with normal multimer structure but a defect in platelet dependent functional parameters did not respond with VWF:RCo. CONCLUSION: Children with VWD type 2 show a variable response to desmopressin depending on the mutation that correlates with the functional defect and the presence or absence as well as the half-life of large VWF multimers. Our data emphasize the usefulness of DDAVP testing even in patients with VWD type 2, possibly with the exception of VWD type 2B.


Assuntos
Desamino Arginina Vasopressina/uso terapêutico , Doenças de von Willebrand/tratamento farmacológico , Criança , Hemostáticos/uso terapêutico , Humanos , Mutação , Fenótipo , Doenças de von Willebrand/genética , Fator de von Willebrand/genética
4.
Ann Hematol ; 81(11): 627-31, 2002 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-12454700

RESUMO

We investigated the efficacy of an antithymocyte globulin/cyclophosphamide preparative regimen prior to allogeneic stem cell transplantation from HLA-identical siblings in patients with severe aplastic anemia. Since 1990, 21 patients, 6 males and 15 females, with a median age of 25 years (range: 7-43) have been enrolled in the protocol consisting of 200 mg/kg cyclophosphamide and 90-120 mg/kg antithymocyte globulin (ATG, rabbit, Fresenius, Bad Homburg, Germany). For further graft-versus-host disease (GVHD) prophylaxis all patients received cyclosporin A and a short course of methotrexate (MTX). Only one patient had a primary graft failure (5%). All other patients engrafted with a leukocyte count >1.0 x 10(9)/l and a platelet count >20 x 10(9)/l after a median of 19 (range: 11-28) and 26 days (range: 13-67), respectively. No late graft failure or relapse was observed. Two patients experienced mild acute GVHD grade I (10%), and one patient developed grade II GVHD (5%). No severe grade III/IV GVHD was observed; 17% of the patients developed limited chronic GVHD. The treatment-related mortality was 14% and mainly due to fungal infection. After a median follow-up of 70 months (range: 2-139), the estimated overall and event-free survival at 10 years for all patients is 86% (95% confidence interval: 70-100%). We conclude that ATG plus cyclophosphamide is an effective conditioning regimen in patients with aplastic anemia undergoing stem cell transplantation with a low treatment-related mortality, resulting in an excellent outcome.


Assuntos
Anemia Aplástica/terapia , Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Transplante de Células-Tronco Hematopoéticas/métodos , Condicionamento Pré-Transplante/métodos , Adolescente , Adulto , Anemia Aplástica/complicações , Anemia Aplástica/mortalidade , Soro Antilinfocitário/administração & dosagem , Protocolos de Quimioterapia Combinada Antineoplásica/toxicidade , Transplante de Medula Óssea/métodos , Transplante de Medula Óssea/mortalidade , Criança , Ciclofosfamida/administração & dosagem , Ciclosporina/administração & dosagem , Feminino , Doença Enxerto-Hospedeiro/tratamento farmacológico , Doença Enxerto-Hospedeiro/patologia , Doença Enxerto-Hospedeiro/prevenção & controle , Transplante de Células-Tronco Hematopoéticas/mortalidade , Humanos , Estudos Longitudinais , Masculino , Metotrexato/administração & dosagem , Micoses/induzido quimicamente , Análise de Sobrevida , Condicionamento Pré-Transplante/mortalidade , Transplante Homólogo/métodos , Transplante Homólogo/mortalidade
5.
Bone Marrow Transplant ; 28(7): 643-7, 2001 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-11704786

RESUMO

We investigated the feasibility and efficacy of a fludarabine-based dose-reduced conditioning regimen followed by stem cell transplantation from related (n = 5) or unrelated HLA-matched donors (n = 7) in 12 patients with high risk MDS, who were not eligible for a standard myeloablative conditioning regimen. The conditioning regimen consisted of fludarabine 30 mg/m(2) daily for 6 days, busulfan 4 mg/kg daily for 2 days and anti-thymocyte globulin (ATG, rabbit) 10 mg/kg daily for 4 days in 11 patients, while one patient received fludarabine, ATG, cyclophosphamide and thiotepa. Graft-versus-host disease prophylaxis consisted of cyclosporine and a short course of methotrexate. The median age of the patients was 53 years (range 37-59). The median percentage of blasts in bone marrow aspirate at transplantation was 15% (range <5% to 35%). Diagnosis at transplant was RA (n = 1), RAEB (n = 5), RAEB-T (n = 5) and sAML (n = 1). A complex karyotype including monosomy 7 was noted in five patients. The reasons for using a dose-reduced conditioning regimen were prior autologous/syngeneic BMT (n = 4), active fungal infection (n = 2) or age/reduced performance status (n = 6). Engraftment was observed in all patients with complete donor chimerism. The incidence of acute GVHD (grade II-IV) was 33%. Eight patients died during follow-up due to relapse (n = 4), liver toxicity (n = 2), aspergillus (n = 1) or aGVHD grade IV (n = 1). After a median follow-up of 19 months, the 2-year estimated disease-free survival is 12% (95% CI: 2-23%) and the overall survival is 26% (95% CI: 4-52%). Fludarabine dose-reduced conditioning prior to allogeneic stem cell transplantation in high risk MDS patients, who were not eligible for standard transplantation, resulted in stable engraftment with complete chimerism, but the toxicity and relapse rate were considerable.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Síndromes Mielodisplásicas/terapia , Condicionamento Pré-Transplante/métodos , Transplante Homólogo , Vidarabina/análogos & derivados , Vidarabina/administração & dosagem , Adulto , Anemia Refratária com Excesso de Blastos/genética , Anemia Refratária com Excesso de Blastos/mortalidade , Anemia Refratária com Excesso de Blastos/patologia , Anemia Refratária com Excesso de Blastos/terapia , Soro Antilinfocitário/administração & dosagem , Soro Antilinfocitário/efeitos adversos , Medula Óssea/patologia , Bussulfano/administração & dosagem , Bussulfano/efeitos adversos , Contagem de Células , Cromossomos Humanos Par 7/genética , Ciclofosfamida/administração & dosagem , Ciclofosfamida/efeitos adversos , Intervalo Livre de Doença , Estudos de Viabilidade , Sobrevivência de Enxerto , Doença Enxerto-Hospedeiro/mortalidade , Doença Enxerto-Hospedeiro/prevenção & controle , Hepatopatia Veno-Oclusiva/etiologia , Hepatopatia Veno-Oclusiva/mortalidade , Histocompatibilidade , Humanos , Infecções/etiologia , Infecções/mortalidade , Cariotipagem , Metotrexato/administração & dosagem , Metotrexato/efeitos adversos , Pessoa de Meia-Idade , Monossomia , Síndromes Mielodisplásicas/genética , Síndromes Mielodisplásicas/mortalidade , Síndromes Mielodisplásicas/patologia , Recidiva , Taxa de Sobrevida , Linfócitos T , Doadores de Tecidos , Condicionamento Pré-Transplante/efeitos adversos , Resultado do Tratamento , Vidarabina/efeitos adversos
6.
Br J Haematol ; 113(4): 1060-71, 2001 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-11442503

RESUMO

We evaluated the cytomegalovirus (CMV) serostatus as a risk factor for survival and treatment-related mortality (TRM) in 125 patients allografted from an unrelated donor between 1994 and 1999. All patients received pretransplant in vivo T-cell depletion using rabbit anti-thymocyte globulin (ATG). Only one patient had primary graft failure and severe grade III/IV graft-versus-host disease occurred in 14% of the patients. The overall survival (OS) at 3 years was 70% for CMV-negative patients (n = 76) and 29% in the seropositive cohort (n = 49) (P > 0.001). In multivariate analyses, CMV seropositivity remained an independent negative prognostic factor for OS (RR: 2.1; CI: 1.2-3.8; P = 0.014), apart from age > 20 years (RR: 2.74; CI: 1.2-3.8; P = 0.004) and late leucocyte engraftment (RR: 2.4; CI: 1.2-4.9; P = 0.015). The TRM for all patients was 27%. Despite monitoring for CMV antigenaemia and preemptive therapy with ganciclovir when reactivation occurred, seropositive patients had a three times higher risk of fatal treatment-related complications than seronegative patients. In multivariate analyses, CMV seropositivity remained the strongest independent negative factor for TRM (RR: 5.3; CI: 1.9-14.6; P = 0.002), followed by age > 20 years (RR: 4.8; CI: 1.3-18.1; P = 0.02) and delayed leucocyte engraftment (RR: 3.6; CI: 1.2-11; P = 0.02). The TRM was identical in seropositive patients with (n = 27) or without (n = 22) CMV reactivation (44% versus 50%). We conclude that CMV seropositivity, despite preemptive ganciclovir therapy and even without reactivation, is a major negative prognostic factor for survival as well as for TRM in unrelated stem cell transplantation using pretransplant in vivo T-cell depletion with ATG.


Assuntos
Soro Antilinfocitário/administração & dosagem , Infecções por Citomegalovirus/complicações , Citomegalovirus , Transplante de Células-Tronco Hematopoéticas/mortalidade , Leucemia/virologia , Condicionamento Pré-Transplante/métodos , Doença Aguda , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Leucemia/terapia , Leucemia Mieloide/terapia , Leucemia Mieloide/virologia , Leucemia Mieloide de Fase Acelerada/terapia , Leucemia Mieloide de Fase Acelerada/virologia , Leucemia Mieloide de Fase Crônica/terapia , Leucemia Mieloide de Fase Crônica/virologia , Leucemia Mielomonocítica Aguda/terapia , Leucemia Mielomonocítica Aguda/virologia , Linfoma/terapia , Linfoma/virologia , Masculino , Pessoa de Meia-Idade , Mieloma Múltiplo/terapia , Mieloma Múltiplo/virologia , Análise Multivariada , Síndromes Mielodisplásicas/terapia , Síndromes Mielodisplásicas/virologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/virologia , Fatores de Risco , Taxa de Sobrevida , Transplante Homólogo
7.
Klin Padiatr ; 211(4): 314-8, 1999.
Artigo em Alemão | MEDLINE | ID: mdl-10472569

RESUMO

BACKGROUND: Sera from healthy individuals contain natural IgM antibodies (anti-NB-Ab) cytotoxic to neuroblastoma (NB) cells. In contrast to healthy children the prevalence of anti-NB-Ab in sera of NB patients is low. Binding of anti-NB-Ab to the NB cell surface leads to activation of complement in vitro. In vivo the injection of the purified IgM fraction from a cytotoxic blood donor results in complete growth arrest of NB xenografts in nude rats. Preliminary results from a phase I/II study to evaluate the pharmacokinetics and side effects of a therapy with anti-NB-Ab are presented here. PATIENTS: Included in this study are patients with NB stage 4 according to INSS with relapse or non-responders to therapy according to the GPOH-NB-therapy protocol. The patients are negative for anti-NB-Ab and are older than one year. METHODS: The therapy is based on a complete exchange of the anti-NB-Ab negative patient serum against serum from an anti-NB-Ab positive ABO-compatible donor by means of plasmapheresis. RESULTS: Up to now, 14 cycles of plasmapheresis have been carried out in 6 NB patients. In 13 of 14 therapy cycles a significant increase in serum toxicity could be observed. Severe side effects have not been seen except a catheter associated thrombosis which was reversible under heparin treatment. After plasmapheresis, pain in the tumor site or regions of metastasis did occur regularly. In some cases temporary elevation of body temperature occurred. One patient had a reduced MIBG uptake after therapy. Tumor necrosis was observed in 2 patients. Three patients showed tumor progress. CONCLUSION: Immunotherapy of NB in children by serum exchange using anti-NB-Ab positive ABO-compatible donor serum is feasible without major side effects and leads to a significant increase of serum toxicity against NB cells.


Assuntos
Imunização Passiva/métodos , Imunoglobulina M/imunologia , Neuroblastoma/imunologia , Neuroblastoma/terapia , Criança , Pré-Escolar , Feminino , Humanos , Imunização Passiva/efeitos adversos , Imunoglobulina M/efeitos adversos , Lactente , Masculino , Plasmaferese/métodos , Análise de Sobrevida
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