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1.
Leukemia ; 20(10): 1751-8, 2006 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-16932344

RESUMO

Acute myeloid leukemia (AML) blasts are immature committed myeloid cells unable to spontaneously undergo terminal maturation, and characterized by heterogeneous sensitivity to natural differentiation inducers. Here, we show a molecular signature predicting the resistance or sensitivity of six myeloid cell lines to differentiation induced in vitro with retinoic acid or vitamin D. The identified signature was further validated by TaqMan assay for the prediction of response to an in vitro differentiation assay performed on 28 freshly isolated AML blast populations. The TaqMan assay successfully predicts the in vitro resistance or responsiveness of AML blasts to differentiation inducers. Furthermore, performing a meta-analysis of publicly available microarray data sets, we also show the accuracy of our prediction on known phenotypes and suggest that our signature could become useful for the identification of patients eligible for new therapeutic strategies.


Assuntos
Antineoplásicos/farmacologia , Resistencia a Medicamentos Antineoplásicos/genética , Leucemia Mieloide/tratamento farmacológico , Leucemia Mieloide/genética , Tretinoína/farmacologia , Doença Aguda , Diferenciação Celular/efeitos dos fármacos , Linhagem Celular Tumoral , Análise por Conglomerados , Bases de Dados Factuais , Regulação Leucêmica da Expressão Gênica/efeitos dos fármacos , Humanos , Leucemia Mieloide/patologia , Metanálise como Assunto , Análise de Sequência com Séries de Oligonucleotídeos , Valor Preditivo dos Testes , Reação em Cadeia da Polimerase Via Transcriptase Reversa , Vitamina D/farmacologia , Vitaminas/farmacologia
3.
J Laparoendosc Adv Surg Tech A ; 12(2): 139-41, 2002 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-12019576

RESUMO

We report a case of ectopic pancreas tissue in the gastric wall that was removed using a minimally invasive approach. The patient was a 46-year-old obese woman who presented with fatigue, weakness, abdominal discomfort, and guaiac-positive stools. Laboratory analysis showed iron deficiency anemia. Preoperative endoscopy revealed a submucosal lesion in the gastric antrum. Intraoperative upper endoscopy clearly located the lesion at the antrum. The lesion was marked with India ink, allowing it to be identified easily at laparoscopy. A laparoscopic wedge resection of the gastric antrum was performed. The patient had an uneventful recovery. We believe that this is a valid treatment option for this benign condition.


Assuntos
Coristoma/cirurgia , Pâncreas , Gastropatias/cirurgia , Feminino , Humanos , Pessoa de Meia-Idade , Antro Pilórico
5.
Thromb Res ; 101(4): 219-30, 2001 Feb 15.
Artigo em Inglês | MEDLINE | ID: mdl-11248282

RESUMO

Low levels of factor X (F.X) were detected in a 4-year-old boy who experienced acute lymphoblastic leukemia and bleeding manifestations. Laboratory data suggested the presence of a dysfunctional F.X molecule. Two novel F.X gene mutations were identified in the proband that was double heterozygous for both: a microdeletion (delC556) in exon VI resulting in a frameshift leading to a termination codon at position 226. This deletion was found in six family members with reduced F.X antigen and activity levels. A second mutation characterised by a G(1344)-->C transversion in exon VIII was detected in the proband resulting in a Lys(408)-->Asn substitution. This latter mutation was present in several asymptomatic family members from the paternal and the maternal side. The proband's sister was homozygous for the Lys(408)-->Asn substitution and exhibited low F.X activity with a normal antigen level. The naturally occurring F.X Lys(408)-->Asn (F.X(K408N)) variant was isolated from plasma of either homozygous or double heterozygous individuals. NH(2)-terminal sequencing of the heavy chain of F.X(K408N) failed to show any sequence abnormality in patients who were also carriers of the delC556, suggesting that this latter lesion accounted for the lack of F.X synthesis. Purified F.X Lys(408)-->Asn had an identical behaviour to normal F.X as judged by SDS-PAGE and immunoblotting. Clotting assay using purified F.X(K408N) and F.X-deficient plasma resulted in a laboratory phenotype similar to that observed in a homozygous subject for F.X Lys(408)-->Asn substitution. This is the first characterisation of a naturally occurring F.X variant with a mutation at the COOH-terminal end of the molecule.


Assuntos
Deficiência do Fator X/genética , Fator X/genética , Mutação , Sequência de Aminoácidos , Substituição de Aminoácidos , Sequência de Bases , Pré-Escolar , Códon de Terminação/genética , DNA/genética , Análise Mutacional de DNA , Deficiência do Fator X/sangue , Deficiência do Fator X/complicações , Feminino , Mutação da Fase de Leitura , Variação Genética , Genótipo , Heterozigoto , Homozigoto , Humanos , Itália , Masculino , Mutação de Sentido Incorreto , Linhagem , Fenótipo , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Deleção de Sequência
6.
Haematologica ; 85(6): 638-46, 2000 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-10870122

RESUMO

BACKGROUND AND OBJECTIVE: Transplantation of hematopoietic stem cells from different sources is being increasingly used to treat a variety of diseases in children. Transplant procedures and indications have changed considerably during recent years. Monitoring of information about these changes is useful for interpretation of nationwide collected data. DESIGN AND METHODS: Since 1985, Centers belonging to the AIEOP (Associazione Italiana Ematologia Oncologia Pediatrica), performing hematopoietic stem cell transplants (HSCT) in children, and members of the AIEOP-Bone Marrow Transplant (BMT) Group annually report data on their transplant activity to the AIEOP-BMT Registry employing specially prepared patient-oriented forms. RESULTS: From January 1985 to December 1998, a total of 2,474 bone marrow (BM), peripheral blood (PB) or umbilical cord blood (CB) transplants were reported: 1,296 (52%) were allogeneic (Allo) and 1,178 (48%) autologous (Auto) transplants. These transplants were performed in 19 Italian Centers on 2,249 patients aged less than 17 years. Among Allo-transplants, 1,198 (92%) were performed using BM progenitor cells, whereas 49 (4%) CB, 42 (3%) were PB, 4 BM plus PB, and 3 BM plus CB allografts; they were performed using HLA-identical sibling donors in 867 cases (67%) and alternative donors (i.e. partially-matched relatives or unrelated donors) in the remaining 429 (33%) cases. Allogeneic transplants were performed on 786 (67%) patients with malignancy and on 395 (33%) patients with non-malignant disorders. In the last 6 years, the number of Allo-transplants per year exceeded that of Auto-transplants. Of the Auto-transplants, 775 (66%) were performed using BM, and 403 (34%) using PB alone or combined with BM hematopoietic stem cells. Indications for Auto-BMT were myelo-lymphoproliferative disorders in 524 (49%) cases, solid tumor in 533 (50%) cases and non-malignant disease in 11 (1%) cases. In the last 5 years, the use of PB for autografts has increased from 7% to 70%. INTERPRETATION AND CONCLUSIONS: These data reflect the development and present status of HSCT in Italy and provide a basis for patient counseling and health care planning.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Adolescente , Transplante de Medula Óssea/estatística & dados numéricos , Criança , Pré-Escolar , Coleta de Dados , Atenção à Saúde , Sangue Fetal , Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Humanos , Itália , Sistema de Registros , Doadores de Tecidos , Transplante Autólogo/estatística & dados numéricos , Transplante Homólogo/estatística & dados numéricos , Resultado do Tratamento
7.
J Pediatr Gastroenterol Nutr ; 27(4): 407-10, 1998 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-9779968

RESUMO

BACKGROUND: In patients with celiac disease, the occurrence of exocrine pancreatic insufficiency has been related to an impairment of the gut-mediated stimulatory effect of the meal on the pancreas. The purpose of this study was to assess the intraduodenal lipase activity in patients with celiac disease by means of the 13C mixed-triglyceride breath test and to monitor pancreatic function after the institution of a gluten-free diet. METHODS: Seventeen untreated patients with celiac disease (mean age, 17.4 +/- 10.5 years) were studied. After an overnight fast, patients were given a standard test meal consisting of 100 g of white bread and 0.25 g of butter per kilogram of body weight, to which 16 mg di-stearyl-13C-octanoyl-glyceride (mixed triglyceride) had been added. Breath samples were taken twice at baseline and at 30-minute intervals for 6 hours after the meal. 13C enrichment in breath was determined by means of Isotope Ratio Mass Spectrometer (IRMS) (ANCA-NT; Europa Scientific, Crewe, UK). Results were expressed as the maximum percentage of 13C recovery per hour at any time, the time to reach peak excretion of 13C, and the percentage of 13C cumulative dose over 6 hours. RESULTS: Mixed-triglyceride breath test results were pathologic in three patients and at the lower limit of the normal range in another patient. In the remaining 13 patients, the results were within normal values. At the 6- and 12-month follow-ups, all patients showed normal intraduodenal lipase activity. CONCLUSIONS: In approximately 24% of patients with celiac disease, the intraduodenal pancreatic lipolytic activity is impaired. The mixed-triglyceride breath test could be used to assess fat maldigestion and to monitor the need for enzyme replacement therapy in such patients.


Assuntos
Testes Respiratórios , Doença Celíaca/enzimologia , Duodeno/enzimologia , Lipase/metabolismo , Triglicerídeos/análise , Adolescente , Adulto , Anticorpos/sangue , Biópsia , Isótopos de Carbono , Doença Celíaca/imunologia , Doença Celíaca/patologia , Criança , Pré-Escolar , Duodeno/patologia , Feminino , Gliadina/imunologia , Humanos , Mucosa Intestinal/patologia , Masculino , Músculos/imunologia
8.
Br J Cancer ; 78(4): 528-33, 1998 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-9716039

RESUMO

The purpose of this study was to evaluate in a phase I-II trial whether low doses of recombinant human interleukin 2 (rHuIL-2) over a prolonged period of time are safe and effective in eradicating or controlling minimal residual disease in children with neuroblastoma given high-dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT). From January 1992 to July 1996, 17 consecutive patients, with either stage IV or relapsed neuroblastoma, were enrolled. Patients received rHuIL-2 after a median time interval (min-max) of 105 days (56-153) after HDCT and ASCT. The protocol consisted of 2 'priming' courses of rHuIL-2 at escalating doses administered intravenously at 72-h intervals, followed by 'maintenance' with 11 monthly and six bimonthly boosting 5-day courses administered subcutaneously on an outpatient basis. At April 1997, 7 out of the 17 patients had completed the treatment schedule, four had discontinued treatment because of toxicity and four because of relapse; the remaining two patients are still on treatment, having completed 15 courses. Expansion of T lymphocytes, together with an increase in both natural killer cells and in activated T lymphocytes was evidenced. After a median (min-max) follow-up time of 30 (16-64) months, 12 out of 17 patients are alive and well. Two patients relapsed and died 14 and 35 months after transplant. Three patients are alive after having relapsed at 41, 21 and 13 months. The actuarial 2-year event-free survival and overall survival are 67% and 92% respectively. Intermittent administration of low doses of rHuIL-2 given for a long period of time is well tolerated and seems capable of controlling minimal residual disease after HDCT and ASCT in children with high-risk neuroblastoma.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/administração & dosagem , Transplante de Células-Tronco Hematopoéticas , Interleucina-2/administração & dosagem , Neuroblastoma/terapia , Criança , Pré-Escolar , Terapia Combinada , Intervalo Livre de Doença , Feminino , Humanos , Lactente , Interleucina-2/efeitos adversos , Masculino , Neuroblastoma/imunologia , Proteínas Recombinantes/administração & dosagem , Transplante Autólogo
9.
J Clin Oncol ; 16(3): 953-65, 1998 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-9508178

RESUMO

PURPOSE: The European Bone Marrow Transplantation (EBMT) Solid Tumor Registry (STR) contains detailed information on children with advanced neuroblastoma who, after standard-dose induction chemotherapy and surgery, received myeloablative megatherapy (MGT) followed by stem-cell transplantation (SCT). This data base was analyzed to identify factors that predict event-free survival (EFS). PATIENTS AND METHODS: Eligibility criteria were stage IV neuroblastoma, age over 1 year at diagnosis, and no relapse before MGT/SCT. Between February 1978 and July 1992, 549 patients were registered by 36 European transplant centers. The median age at diagnosis was 36 months (range, 13 to 216 months) and the male-female ratio was 1:45. Before MGT, 157 patients were in complete remission (CR), 156 in very good partial remission (VGPR), and 208 in partial remission (PR), whereas 24 had had only a minor response (MR). One hundred ten of 546 patients had undergone two successive MGT procedures. The median observation time was 60 months (range, 12 to 187 months). RESULTS: Actuarial EFS is 26% at 5 years. Multivariate analysis by the Cox proportional hazards regression model included 529 patients with complete data sets. After adjustment for treatment duration before MGT and double MGT procedures, two adverse, independent risk factors that influenced EFS were identified: (1) persisting skeletal lesions before MGT as defined by technetium (99TC) scans and/or meta-iodobenzylguanidine (mIBG) scans (P = .004) and (2) persisting bone marrow involvement before MGT (P = .03). CONCLUSION: After induction treatment, persisting skeletal disease as defined above and persisting bone marrow involvement may be predictive of a particularly poor outcome. Physicians may consider this an additional important tool to decide the patient's management.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas , Neuroblastoma/terapia , Adolescente , Neoplasias Ósseas/secundário , Criança , Pré-Escolar , Terapia Combinada , Feminino , Humanos , Lactente , Masculino , Análise Multivariada , Neoplasia Residual , Neuroblastoma/mortalidade , Neuroblastoma/secundário , Prognóstico , Modelos de Riscos Proporcionais , Indução de Remissão , Estudos Retrospectivos , Fatores de Risco , Análise de Sobrevida
10.
Pediatr Allergy Immunol ; 8(4): 190-3, 1997 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-9553984

RESUMO

The aim of the present study was to evaluate the prevalence of soy allergy (positive skin test and positive challenge test) in a large cohort of atopic children, many of them soy fed early in life for several months. In order to investigate the prevalence of soy allergy, two groups of children were enrolled into the study. The first group comprised a cohort of 505 children with personal history suggestive of food allergy. The second group included 243 children born of atopic parents, who had been soy protein formula fed for the first six months of life for the prevention of cow's milk allergy and who had been prospectively followed up, from birth to 5 years. As regards the prevalence of soy allergy in the cohort of children suffering from allergic disease: 31/505 children (6%) had positive skin prick test to soy, however only six of the 31 children with positive skin prick test to soy had positive challenge test to soy. With regard to the prevalence of soy allergy in the children who had been soy protein formula fed in the first six months of life (second group): 14/243 children (6%) had positive skin prick test to soy, but the double blind placebo control oral food challenge to soy was positive in only one of these 14 children. In conclusion documented soy allergy is not common in atopic children.


Assuntos
Alérgenos/imunologia , Hipersensibilidade Alimentar/imunologia , Proteínas de Soja/imunologia , Pré-Escolar , Feminino , Hipersensibilidade Alimentar/epidemiologia , Humanos , Lactente , Masculino , Variações Dependentes do Observador , Prevalência , Estudos Prospectivos , Testes Cutâneos/métodos , Proteínas de Soja/administração & dosagem , Proteínas de Soja/efeitos adversos
11.
Bone Marrow Transplant ; 18 Suppl 2: 4-7, 1996 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-8932789

RESUMO

During the "2nd International Course on Bone Marrow Transplantation in Children" a multiple choice questionnaire on bone marrow transplant indications for children with acute leukemias was distributed with the aim of achieving a consensus. The answers obtained from the twenty representatives of fourteen European countries during the meeting were analyzed and assigned to one of the following groups: I. definitive indication: when more than 75% participants were in favour; II. acceptable indication: when 50% to 74% participants were in favour; III. requires further investigation: when 25% to 49% participants were in favour; IV. no indication: when less than 24% participants were in favour. In acute lymphoblastic leukemia the following circumstances were considered a definitive indication for allogeneic bone marrow transplant (BMT) from a matched sibling donor (MSD): infancy, "high risk" (HR) patients in 1st complete remission (CR1); CR2 patients after an early bone marrow relapse (defined as a relapse occurring up to six months after stopping therapy). Patients experiencing an early meningeal relapse and CR2 patients after a late relapse (defined as a relapse occurring later than six months after stopping therapy) were considered an acceptable indication. Further investigation was required in order to better define the role of BMT for patients experiencing an early isolated testicular relapse. If a MSD is not available, HR patients in CR1 and CR2 patients, after an early bone marrow relapse, were considered a definite indication for a matched unrelated donor (MUD). This latter group was considered an acceptable indication for a haploidentical BMT if a MUD was not available. Further investigation was required to better define the role of autologous bone marrow transplant (ABMT) for patients experiencing an early extramedullary relapse and for HR patients in CR1 all of whom lacked MSD's. In acute myeloblastic leukemia (AML), CR2 patients were considered a definitive indication and CR1 patients were considered an acceptable indication for BMT from a MSD. CR2 patients were considered a definitive indication for ABMT and CR1 patients an acceptable indication in cases lacking a MSD. AML was not considered an indication for MUD BMT.


Assuntos
Transplante de Medula Óssea , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Humanos , Lactente , Recidiva , Resultado do Tratamento
13.
Bone Marrow Transplant ; 18 Suppl 2: 43-6, 1996 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-8932798

RESUMO

The development of chemotherapy in childhood ALL has been the leader of the success story of paediatric oncology. At least 2/3 of the children can be cured nowadays at the first attempt of treatment. From the remaining again 1/3 can be treated successfully for the relapse of their disease with conventional therapeutic strategies. This means, however, that there is no chance for cure with chemotherapy alone for 20 to 25% of the children. BMT has been shown for a long time to be an alternative therapy especially in those cases in which conventional chemotherapy fails. In spite of the fact that many children with ALL have been transplanted during recent years there is still no general agreement on the question which children need BMT. However a few statements can be made: The value of ABMT in ALL is probably not better than that of chemotherapy alone. In 1st CR a group of children can be defined, which might benefit from BMT. In 2nd CR the value of chemotherapy depends very much from the duration of 1st remission. Allogeneic BMT is the only chance for cure in very early relapses, superior to chemotherapy in early and late relapses and possibly equal to chemotherapy in very late relapses. The paper tries to summarise our current knowledge about the situation.


Assuntos
Transplante de Medula Óssea , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Criança , Pré-Escolar , Humanos , Lactente , Leucemia-Linfoma Linfoblástico de Células Precursoras/patologia , Recidiva , Resultado do Tratamento
14.
Bone Marrow Transplant ; 18 Suppl 2: 49-52, 1996 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-8932799

RESUMO

The Austrian-German-Italian (AGI) Pediatric Bone Marrow Transplantation Registry includes now data of 520 patients grafted for acute myeloid leukemia (AML) by autologous and allogeneic stem cells. In first complete remission (CR1) 145 patients with allografts had a possibility of event free survival (pEFS) of 0.57 and 140 patients with autografts a pEFS of 0.46. In second complete remission (CR2) autografted patients (n = 70) had a pEFS of 0.36 and allogeneic patients (n = 41) of 0.34. Patients with no CR went worse (allogeneic pEFS 0.15 n = 37; autologous pEFS 0.17 n = 6). Therefore, from the European data the following conclusions can be drawn: neither in CR1 nor in CR2 any statistical advantage for a particular transplantation type can be found. To assess the value of transplantation with family mismatch donors or matched unrelated donors (MUD) basing on these data pool is not possible. The following decisions should be reached: 1) BMT for AML with mismatched or unrelated donors should be done only within cooperative European trials. 2) Generally accepted definitions of indications for BMT in AML are needed. 3) Study protocols concerning conditioning are necessary.


Assuntos
Transplante de Medula Óssea/métodos , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Criança , Pré-Escolar , Europa (Continente) , Humanos , Lactente , Transplante Autólogo , Transplante Homólogo , Resultado do Tratamento
18.
Immunol Today ; 17(11): 511-6, 1996 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-8961627

RESUMO

X-linked hyper-IgM syndrome (X-HIM) is an immunodeficiency caused by mutations in the gene encoding the CD40 ligand (CD40L). A database (CD40Lbase) of CD40L mutations has now been established, and the resultant information, together with other mutations reported elsewhere in the literature, is presented here.


Assuntos
Antígenos CD40/genética , Bases de Dados Factuais , Hipergamaglobulinemia/genética , Imunoglobulina M/genética , Glicoproteínas de Membrana/genética , Mutação , Cromossomo X , Ligante de CD40 , Ligação Genética , Humanos , Ligantes
19.
Bone Marrow Transplant ; 18(1): 125-30, 1996 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-8832005

RESUMO

This study reports a large cooperative experience in myeloablative therapy and bone marrow rescue undertaken to define better the outcome of children with disseminated neuroblastoma after megatherapy. Between 1984 and 1993, 135 children underwent myeloablative therapy with bone marrow transplantation (BMT) in nine Italian Centres. One hundred and seventeen children received unpurged autologous BMT, five allogeneic BMT and 13 peripheral blood progenitor cells as rescue. Of these 135 children, 57 were in 1st CR, 11 in 2nd or subsequent CR, 42 in 1st PR, and 25 had more advanced disease. Twelve children (9%) died of toxicity, 86 relapsed or progressed at 1-68 months (median 7 months) and 80 of these subsequently died of progressive disease. Forty-three children are still alive with 37 in continuous remission at a median of 65 months (30-123 months) after BMT. Overall and disease-free survival at 8 years are 28.5% (s.e. 4.3) and 26% (s.e. 4), respectively. Disease-free survival is 34.6% (s.e. 6.7) for the patients grafted in 1st complete remission, 23.6% (s.e. 6.6) for patients grafted in 1st partial remission, 36.4% (s.e. 14.5) for patients grafted in 2nd or subsequent CR, and 8% (5.4) for patients with advanced disease. We conclude these data confirm that early toxicity of myeloablative therapy is manageable and that myeloablative therapy with bone marrow rescue may contribute to an improved long-term survival of children with disseminated neuroblastoma but the objective of cure of all patients remains distant.


Assuntos
Transplante de Medula Óssea , Neuroblastoma/terapia , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Medula Óssea/estatística & dados numéricos , Doença Hepática Induzida por Substâncias e Drogas , Criança , Pré-Escolar , Terapia Combinada , Intervalo Livre de Doença , Feminino , Humanos , Lactente , Recém-Nascido , Infecções/etiologia , Infecções/mortalidade , Itália/epidemiologia , Hepatopatias/mortalidade , Masculino , Melfalan/administração & dosagem , Neuroblastoma/tratamento farmacológico , Neuroblastoma/mortalidade , Neuroblastoma/patologia , Neuroblastoma/radioterapia , Sistema de Registros , Análise de Sobrevida , Taxa de Sobrevida , Condicionamento Pré-Transplante/efeitos adversos , Resultado do Tratamento , Vincristina/administração & dosagem , Irradiação Corporal Total/efeitos adversos
20.
Bone Marrow Transplant ; 14(1): 37-46, 1994 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-7951119

RESUMO

A case control study was performed to investigate the potential advantage of allogeneic bone marrow transplantation in advanced or poorly responding neuroblastoma first remission patients using the European BMT Solid Tumor Registry. Seventeen allogeneic and 34 autologous bone marrow transplantation (BMT) cases were matched based on a number of prognostic factors including age, sex, prior treatment duration, pre-graft response status and bone and BM involvement before BMT. Only single BMT procedures are included. The median age at diagnosis was 47 months (range 18-113 months). The median follow-up time since BMT is 58 months (range 13-133 months). The only significant prognostic factor within the allogeneic BMT (p = 0.012) and autologous BMT groups (p = 0.025) was residual skeletal disease before BMT, detected by mIBG in 86% of the cases. However, the progression-free survival was not significantly different: 35% and 41% at 2 years, respectively. Only half of the allogeneic BMT patients had developed graft-versus-host disease (GVHD): 7 of 9 grade I-II and only 2 of 9 grade IV. The median donor age was very young with 74 months (range 20-240 months) and 10 of 17 were sex matched. Thus absence of GVHD risk factors in young children could be the major obstacle in achieving an anti-tumor effect with allogeneic BMT in neuroblastoma.


Assuntos
Transplante de Medula Óssea , Neuroblastoma/terapia , Transplante de Medula Óssea/efeitos adversos , Estudos de Casos e Controles , Criança , Pré-Escolar , Terapia Combinada , Europa (Continente) , Feminino , Doença Enxerto-Hospedeiro/etiologia , Humanos , Lactente , Masculino , Prognóstico , Sistema de Registros , Estudos Retrospectivos , Transplante Autólogo , Transplante Homólogo
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