Assuntos
Antimetabólitos Antineoplásicos/uso terapêutico , Azacitidina/análogos & derivados , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Azacitidina/uso terapêutico , Criança , Decitabina , Feminino , Humanos , Leucemia-Linfoma Linfoblástico de Células Precursoras/patologia , Resultado do TratamentoRESUMO
The authors report the results of 58 children with ALL in 2CR after related (n = 31) or unrelated (n = 27) AHSCT. Characteristics at diagnosis and initial and after relapse antileukemic treatment were similar in the related donor (RD) and the unrelated donor (UD) groups. Conditioning consisted of TBI/CY +/- VP-16 for patients > or = 3 years old (n = 43) and Bu/CY for the rest. Median recipient age was 8 years (range 1-17) in the RD and 9 years (range 3-14) in the UD group. Median follow-up was 54 months (range 24-80) and 52 months (range 22-85) in the RD and the UD groups repectively. The 5-year EFS probability was 43 +/- 9% for the RD group and 36 +/- 9% in the UD group (p = .25). The transplant-related mortality was 16% in the RD and 37% in the UD group (p = .016). In the RD group 36.7% of patients relapsed versus 18.6% in the UD group (p = .05). GvHD associated with organ failure or infection caused most of the transplant-related deaths in both groups. Survivor quality of life for both groups was good (Lansky score < or = 90).
Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Criança , Pré-Escolar , Terapia Combinada , Feminino , Doença Enxerto-Hospedeiro/mortalidade , Doença Enxerto-Hospedeiro/prevenção & controle , Transplante de Células-Tronco Hematopoéticas/mortalidade , Humanos , Lactente , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/imunologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Qualidade de Vida , Recidiva , Indução de Remissão , Taxa de Sobrevida , Condicionamento Pré-Transplante , Transplante Homólogo , Resultado do TratamentoRESUMO
Allogeneic stem cell transplantation is the only curative treatment for Wiskott-Aldrich syndrome. The authors retrospectively analyzed the outcome with this procedure in 13 patients with severe Wiskott-Aldrich syndrome transplanted in 5 Spanish centers from 1989 to 2006. A patient was transplanted twice from the same donor due to a late engraftment failure. Age at transplant ranged from 7 to 192 months (median 30 months). There were 10 matched donors (3 related and 7 unrelated), 2 mismatched unrelated, and 1 haploidentical. Conditioning regimen consisted of busulfan and cyclophosphamide (BuCy) in 11 cases and fludarabine and melfalan (1) or BuCy (1). ATG was added in transplants from non-genetically matched donors. GvHD prophylaxis consisted of cyclosporine and methotrexate in most patients plus T-cell depletion in the haploidentical HSCT. Nine of the 13 transplanted patients are alive with complete clinical, immunologic, and hematologic recovery 8-204 months (median 101 months) after HSCT. Eight surviving patients had been transplanted from matched donors (3 related and 5 unrelated) and 1 from a haploidentical donor. Four patients died, 2 transplanted from matched donors (1 from acute GvHD and organ failure, 1 from a lymphoproliferative disorder after a second transplant), and 2 transplanted from mismatched unrelated donors (1 from acute GvHD and organ failure, 1 from graft failure and infection). Allogeneic hemopoietic stem cell transplantation must be utilized in all patients with severe Wisckott-Aldrich syndrome, using the most suitable graft variant for each patient.
Assuntos
Transplante de Células-Tronco Hematopoéticas/estatística & dados numéricos , Síndrome de Wiskott-Aldrich/cirurgia , Soro Antilinfocitário/uso terapêutico , Bussulfano/uso terapêutico , Criança , Pré-Escolar , Ciclofosfamida/uso terapêutico , Ciclosporina/uso terapêutico , Doença Enxerto-Hospedeiro/mortalidade , Doença Enxerto-Hospedeiro/prevenção & controle , Antígenos HLA/genética , Antígenos HLA/imunologia , Haplótipos , Transplante de Células-Tronco Hematopoéticas/mortalidade , Histocompatibilidade , Humanos , Imunossupressores/uso terapêutico , Lactente , Doadores Vivos , Depleção Linfocítica , Masculino , Melfalan/uso terapêutico , Insuficiência de Múltiplos Órgãos/mortalidade , Complicações Pós-Operatórias/mortalidade , Reoperação , Estudos Retrospectivos , Espanha/epidemiologia , Linfócitos T , Condicionamento Pré-Transplante , Transplante Homólogo/mortalidade , Transplante Homólogo/estatística & dados numéricos , Resultado do Tratamento , Vidarabina/análogos & derivados , Vidarabina/uso terapêutico , Síndrome de Wiskott-Aldrich/epidemiologiaRESUMO
We present a retrospective study of long-term outcome and predictive factors of survival and relapse in 219 paediatric patients with acute lymphoblastic leukaemia (ALL) in second remission. They received allogeneic (allo) or autologous (auto) haemopoietic cell transplantation (HCT) depending on the availability of a matched sibling donor. The probability of event-free survival (EFS) for the total patient group was 0.35+0.03 at 14 years. No significant differences were observed for EFS between allo- and auto-HCT: 0.39+0.05 vs 0.32+0.04 (P=0.43). A better EFS was seen in patients with a late relapse (LR) (P=0.06 and 0.02, for allogeneic and autologous respectively). Significantly better EFS was observed in allo-HCT patients under 10 years of age and in auto-HCT patients with leukocytes at diagnosis below 25 x 109/l and late relapse. Predictive factors of failure in both groups were early relapse (ER), medullary relapse and age over 10 years. The probability of relapse (RP) for the total group of patients was 0.57+0.03, and it was significantly higher in auto-HCT patients: 0.65+0.04 vs 0.42+0.06 (P=0.002). Factors predictive for relapse were medullary and early relapse, auto-HCT and WBC >25 x 109/l at diagnosis.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Humanos , Lactente , Recém-Nascido , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Recidiva , Estudos Retrospectivos , Transplante Autólogo , Resultado do TratamentoRESUMO
The authors retrospectively analyzed the long-term outcome of 67 patients over 1 year of age at diagnosis with high-risk neuroblastoma (stage 4 or stage 3 with N-myc amplification) who were treated with megatherapy and stem cell rescue from 1984 to 1998. Median age at transplant was 4 years (range 1.6-15 years). The source of cells was peripheral stem cells in 29 and bone marrow in 38 patients. In 12 patients, an in vitro purging of bone marrow harvest was performed. Most patients were conditioned with melphalan, BCNU, and VM-26. After transplant 19 patients received complementary treatment with IL-2 (16) or 13-cis-retinoic acid (3). Six patients (8%) died from transplant-related toxicity and 39 from disease progression. Three patients were alive with active disease at the time of analysis. Nineteen patients are alive and disease-free at a median follow-up of 104 months. Five-year event-free survival is 0.30. Survival of patients who received a purged graft was not significantly better than the rest. Post-transplant complementary treatment significantly improved overall and event-free survival (p = .01 and p = .04, respectively).
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas , Neuroblastoma/terapia , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Neuroblastoma/mortalidade , Estudos Retrospectivos , Transplante AutólogoRESUMO
This multicenter study was designed to evaluate whether allo-PBPCT provides some advantages, if any, over BMT in terms of engraftment kinetics, acute and chronic GVHD incidence, TRM, relapse incidence and survival in acute lymphoblastic leukemia patients (ALL). From January 1995 to December 1999, 67 ALL patients (34 in the PBPCT group and 33 in the BMT group) were included in this study. Median age for both groups was 8 years (range, 1-18). There were 24 patients in first or second CR in the PBPCT group and 28 such patients in the BMT group. Preparatory regimens were TBI-based in 26/34 in the PBPC group and 25/33 in the BMT group. GVHD prophylaxis was CsA alone in 38 patients (18 PBPCT vs 20 BMT) and CsA plus short Mtx in 29 (16 PBPCT vs 13 BMT). Engraftment was achieved in all cases. Median days to neutrophil recovery was 10 (range, 7-18) after PBPCT vs 14 (range, 9-21) after BMT (P < 0.0001). Platelet engraftment (>50 x 10(9)/l) was also faster for PBPCT patients (median 13 days, range, 9-40 vs 23 days, range, 15-165) (P < 0.0001). Acute GVHD grade II-IV incidence was similar in both groups (46.4 +/- 8.8% vs 42.7 +/- 8.6%) (P = 0.45). Probability of chronic GVHD was 50.6 +/- 12.2% after PBPCT vs 27.8 +/- 9.2% after BMT (P = 0.1). Probability of relapse was similar (28.7 +/- 9.2% for PBPCT vs 27.1 +/- 8.2% for BMT) (P = 0.89). There were eight patients who died from transplant-related complications after PBPCT vs 5 after BMT (P, NS). With a median follow-up of 25 months the event-free survival probability was 53 +/- 8.9% for PBPCT vs 54.9 +/- 9.7% for BMT (P = 0.54). Using PBPC for allogeneic transplantation in childhood ALL results in faster hematopoietic recovery compared to BM, with a similar incidence of aGVHD, TRM, relapse and disease-free survival. However, the issue of cGVHD remains unresolved.
Assuntos
Transplante de Medula Óssea , Transplante de Células-Tronco de Sangue Periférico , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Adolescente , Adulto , Transplante de Medula Óssea/efeitos adversos , Transplante de Medula Óssea/mortalidade , Criança , Pré-Escolar , Família , Feminino , Sobrevivência de Enxerto , Doença Enxerto-Hospedeiro , Histocompatibilidade , Humanos , Incidência , Lactente , Cinética , Masculino , Análise por Pareamento , Transplante de Células-Tronco de Sangue Periférico/efeitos adversos , Transplante de Células-Tronco de Sangue Periférico/mortalidade , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Recidiva , Transplante Homólogo/efeitos adversos , Transplante Homólogo/mortalidade , Transplante Isogênico/efeitos adversos , Transplante Isogênico/mortalidadeRESUMO
Recently advances have been made in the treatment of acute leukemia in children, it is now possible to cure more than 70% of children with acute lymphoblastic leukemia. With the introduction of more intensive chemotherapy regimens in patients at higher risk of relapse and the identification of cases that could be less intensely treated to diminish long-term toxicity, it could be possible to improve these excellent results. In contrast, pediatric acute myeloid leukaemia seems to be a more heterogeneous disease and its response to conventional chemotherapy is not as uniform. Introduction of new and more efficacious therapies is necessary to improve the poor outcome, especially among patients with high-risk features.
Assuntos
Leucemia/terapia , Doença Aguda , Transplante de Medula Óssea , Criança , Terapia Combinada , Humanos , Leucemia/tratamento farmacológico , RecidivaRESUMO
PURPOSE: Infants with acute leukemia have a poor prognosis when treated with conventional chemotherapy. It is still unknown if stem-cell transplantation (SCT) can improve the outcome of these patients. In the present study, we review our experience with SCT in infant acute leukemia to clarify this issue. PATIENTS AND METHODS: We report the results of 26 infants who were submitted to a SCT for acute leukemia. There were 15 cases of acute myeloid leukemia and 10 cases of acute lymphoid leukemia. One patient had a bilineal leukemia. Twenty-two patients were in their first complete response (CR1), three were in their second CR, and one was in relapse. Eight patients were submitted to allogeneic SCT, and 18 underwent autologous SCT. RESULTS: With a median follow-up of 67 months, the 5-year overall survival and disease-free survival (DFS) are 64% (SE = 9%) and 63% (SE = 10%), respectively. Autologous and allogeneic SCT offered similar outcome. There was not any transplant-related mortality, and all deaths were caused by relapse in the first 6 months after SCT. In multivariate analysis, the single factor associated with better DFS was an interval between CR1 and SCT of less than 4 months (P: <.025). CONCLUSION: SCT is a valid option in the treatment of infant acute leukemia, and it may overcome the high risk of relapse with conventional chemotherapy showing very reduced toxicity. This study suggests that SCT should be performed in CR1 in the early phase of the disease.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide/terapia , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Doença Aguda , Análise de Variância , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Terapia Combinada , Intervalo Livre de Doença , Feminino , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Lactente , Recém-Nascido , Leucemia Mieloide/tratamento farmacológico , Modelos Logísticos , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Estudos Retrospectivos , Resultado do TratamentoRESUMO
Recently, a consensus International Prognostic Scoring System (IPSS) for predicting outcome and planning therapy in the myelodysplastic syndromes (MDS) has been developed. However, the intermediate-risk cytogenetic subgroup defined by the IPSS includes a miscellaneous number of different single abnormalities for which real prognosis at present is uncertain. The main aims of this study were to evaluate in an independent series the prognostic value of the IPSS and to identify chromosomal abnormalities with a previously unrecognized good or poor prognosis in 640 patients. In univariate analyses, cases with single 1q abnormalities experienced poor survival, whereas those with trisomy 8 had a higher risk of acute leukaemic transformation than the remaining patients (P = 0.004 and P = 0.009 respectively). Patients with single del(12p) had a similar survival to patients with a normal karyotype and showed some trend for a better survival than other cases belonging to the IPSS intermediate-risk cytogenetic subgroup (P = 0.045). Multivariate analyses demonstrated that IPSS cytogenetic prognostic subgroup, proportion of bone marrow blasts and haemoglobin level were the main prognostic factors for survival, and the first two characteristics and platelet count were the best predictors of acute leukaemic transformation risk. A large international co-operative study should be carried out to clarify these findings.
Assuntos
Aberrações Cromossômicas , Síndromes Mielodisplásicas/genética , Adulto , Idoso , Transformação Celular Neoplásica , Feminino , Humanos , Fragmentos Fab das Imunoglobulinas , Incidência , Leucemia Mieloide/genética , Leucemia Mieloide/mortalidade , Masculino , Pessoa de Meia-Idade , Síndromes Mielodisplásicas/mortalidade , Prognóstico , Taxa de SobrevidaRESUMO
This study evaluates the outcome of myeloablative chemo-radiotherapy and autologous stem cell transplantation (ASCT) in children with Hodgkin's disease (HD). Twenty children aged 5 to 18 years (median 10.8 years) at diagnosis, with relapsed, refractory or very poor prognosis HD, underwent ASCT in eight hospitals of our country. Status at transplant was: second complete remission (CR2): n = 12; further CR (CR >2): n = 3, partial remission (PR): n = 2, relapse: n = 2 and first CR (CR1): n = 1. Eighteen patients received chemotherapy-based conditioning regimens: cyclophosphamide, carmustine and etoposide (CBV): 11 (55%), carmustine, etoposide, cytarabine and melphalan (BEAM): 5, other: 2; and two patients were conditioned with TBI/Cy. Peripheral blood (PB) was the source of progenitor cells in 12 patients, BM in seven, and BM plus PB, in one. All patients engrafted. One patient died of sepsis and multiorgan failure at day 28 after transplantation. All four patients with measurable disease (PR or relapse) at transplantation attained complete remission. Five patients relapsed 5-34 months after transplant (median: 11 months). Eighteen children remain alive with a median survival time of 40 months. The projected 5-year overall survival and event-free survival (EFS) rates were 0.95 and 0.62. High-dose therapy with stem cell rescue can lead to durable remissions in children with advanced HD. Bone Marrow Transplantation (2000) 25, 31-34.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Células-Tronco Hematopoéticas , Doença de Hodgkin/terapia , Radioterapia , Adolescente , Criança , Pré-Escolar , Terapia Combinada , Feminino , Humanos , Masculino , Agonistas Mieloablativos/uso terapêutico , Estudos Retrospectivos , Transplante Autólogo , Resultado do TratamentoRESUMO
Molecular rearrangements of the MLL gene at the 11q23 region have been identified in most cases of infant leukemia, regardless of the phenotype. We present a case of acute myeloid leukemia which coexpressed myeloid and lymphoid markers in a 12-month-old girl. Karyotype analysis revealed the presence of a thus far unreported translocation t(10;11)(p13;p15). Although no 11q23 abnormalities were cytogenetically detectable, an MLL gene molecular rearrangement was found.
Assuntos
Cromossomos Humanos Par 10 , Cromossomos Humanos Par 11 , Proteínas de Ligação a DNA/genética , Rearranjo Gênico , Leucemia Mieloide/genética , Proto-Oncogenes , Fatores de Transcrição , Translocação Genética , Doença Aguda , Feminino , Histona-Lisina N-Metiltransferase , Humanos , Lactente , Proteína de Leucina Linfoide-MieloideRESUMO
BACKGROUND: Identification of RBC pyruvate-kinase (PK) gene mutations by polymerase chain reaction (PCR) and single strand conformation polymorphism (SSCP) followed by PK gene sequencing in positive cases has been assessed and the results obtained with a preliminary study of 15 unrelated patients of Spanish origin are presented. PATIENTS AND METHODS: Patients have been classified into two different groups: group 1, propositus (15 cases), and group 2, relatives of the patients included in group 1 (10 males and 5 females). In group 1, a PCR was followed by SSCP and sequencing, and in group 2, the PCR was followed by digestion with specific restriction endonucleases (PCR-ER). RESULTS: Group 1: from 15 patients included in the study 2 were identified as homozygous, 4 as heterozygous and 9 as compound heterozygous. In this group, were identify 26 affected alleles with 11 different mutations: T1456 10 alleles (38.6%), T721 3 alleles (11.6%), A1010, C514, C1015 and T1223 2 alleles (7.7%), and C1070, A1291, T1508, A1595 y T1675 one allele. Relatives from 8 out of 15 patients from group 1 showed the following pattern: homozygous (one case), heterozygous (10 cases), compound heterozygous (2 cases) and normal (2 cases). CONCLUSIONS: SSCP procedure followed by direct gene sequencing in positive cases is fast and simple enough to allow the identification of PK deficient variants, avoiding the need of biochemical characterisation of semipurified deficient enzyme, which is more cumbersome and time consuming. In addition, the PCR-ER method is a very useful tool for screening of the most frequent molecular variants, as well as, for the detection of the carrier condition of this enzymopathy (family studies).
Assuntos
Anemia Hemolítica/genética , Eritropoese/genética , Piruvato Quinase/deficiência , Piruvato Quinase/metabolismo , Aminoácidos/genética , Anemia Hemolítica/enzimologia , Doença Crônica , Feminino , Genótipo , Hematologia , Humanos , Masculino , Nucleotídeos/genética , Mutação Puntual/genética , Reação em Cadeia da Polimerase , Polimorfismo Conformacional de Fita Simples , Análise de Sequência de DNA , Sociedades Médicas , EspanhaRESUMO
BACKGROUND AND OBJECTIVE: To compare the estimated survival and disease-free survival between children with Ph1-positive chronic myeloid leukemia treated with allogeneic bone marrow transplantation or conventional chemotherapy. DESIGN AND METHODS: In this retrospective study we compared the results obtained in a group of 14 children who received allogeneic bone marrow transplantation (BMT) between 1983 and 1993, and another group of 27 children treated with busulfan, hydroxyurea or alpha-interferon during the same time period. Patients were transplanted at a median of 7 months from diagnosis and all except one were in their first chronic phase. Conditioning consisted in total body irradiation and cyclophosphamide in 12 cases, and busulfan was added in two. RESULTS: Of the 14 patients treated with BMT, two died of transplant-related complications and two relapsed 18 and 48 months after the BMT. Ten children remain alive and disease free at a median follow up of 60 months. The probability of DFS at 5 years is 70%. Of the 27 patients treated with chemotherapy, 22 have died at a median of 36 months from diagnosis. The probability of survival at 5 years is 5% versus 83% for the BMT group (p = 0.001). INTERPRETATION AND CONCLUSIONS: Allogeneic BMT is a safe and very effective treatment for Ph-positive CML in children. Patients who have an HLA-identical sibling donor must receive a transplant as soon as possible after being diagnosed.
Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Medula Óssea , Leucemia Mielogênica Crônica BCR-ABL Positiva/terapia , Adolescente , Transplante de Medula Óssea/mortalidade , Bussulfano/administração & dosagem , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Seguimentos , Humanos , Hidroxiureia/administração & dosagem , Interferon-alfa/administração & dosagem , Leucemia Mielogênica Crônica BCR-ABL Positiva/tratamento farmacológico , Tábuas de Vida , Masculino , Indução de Remissão , Estudos Retrospectivos , Espanha/epidemiologia , Análise de Sobrevida , Condicionamento Pré-Transplante , Resultado do TratamentoRESUMO
The PK-LR gene has been studied in 12 unrelated patients with red cell pyruvate kinase deficiency and hereditary nonspherocytic haemolytic anaemia (CNSHA). The entire codifying region of the R-type PK gene and the flanking intronic regions were analysed by single-stranded conformation polymorphism (SSCP) followed by direct sequencing of abnormal DNA. 10 different mutations were identified in 22/24 alleles at risk. Eight of these were missense mutations that caused the following single amino acid changes: G514C (172Glu-Gln), G1010A (337Arg-Gln), G1015C (339Asp-Gln), T1070C (357Ile-Thr), C1223T (408Thr-Ile), G1291A (431Ala-Thr), C1456T (486Arg-Trp) and G1595A (532Arg-Gln). Two were nonsense mutations: G721T (241Glu-Stop) and C1675T (559Arg-Stop). 7/22 alleles demonstrated the same C1456 --> T mutation. The study of the polymorphic site at nucleotide (nt) 1705 performed in all cases disclosed a 1705 C/C mutation in 10 and a 1705 A/C mutation in three. This is the first report on the presence of several different L-type PK gene mutations within Spanish population. Furthermore, from the PK gene mutations found, six were unique and not previously described (1015C, 1070C, 1223T, 1291A, 1595A and 1675T) and one (C1456T) seems to be predominant in Spain. Interestingly, no case with the 1529A mutation commonly found in Northern European populations was present here.
Assuntos
Anemia Hemolítica Congênita não Esferocítica/genética , Piruvato Quinase/genética , Adolescente , Adulto , Anemia Hemolítica Congênita não Esferocítica/sangue , Anemia Hemolítica Congênita não Esferocítica/enzimologia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Mutação , Mutação Puntual , Reação em Cadeia da Polimerase , Polimorfismo Conformacional de Fita Simples , Piruvato Quinase/deficiência , Análise de Sequência de DNA , EspanhaRESUMO
A quality-of-life questionnaire study was administered in a group of 98 disease-free survivors more than 3 years after BMT. All participants were over the age of 17 years at the time of the survey. The transplants were performed between 1981 and 1993 in eight Spanish hospitals. Eighty-three percent of patients had undergone BMT for neoplastic disease. Seventy-three per cent received an allogeneic bone marrow transplantation. A modified version of a questionnaire applied in Stanford Hospital to evaluate quality of life in adults after BMT was used. A single investigator was responsible for interviewing all subjects by telephone. We compare these results with the same questionnaire applied in a control group of 58 healthy subjects of similar age. The most significant results were: BMT patients valued their quality of life more highly than the control group. The mean score for global quality of life was 8.19+/-0.17 in BMT group as compared to 7.54+/-0.13 in control group (P=0.0013). Studies were cited as the major concern in both groups: 24% in BMT group and in 69% in control group (C.I. 95%=0.59 to 0.30). The patients in the BMT group considered they had fewer problems in comparison with the control group regarding interpersonal relationships with family members and friends, sleep, depression and leisure possibilities. However, they considered they had more problems concerning their physical appearance, studies and work possibilities than their peers. Considerations regarding weight, height, sexual functioning, anxiety, tendency to suffer illness and problems with insurance were similar in both groups.
Assuntos
Transplante de Medula Óssea/psicologia , Qualidade de Vida , Adolescente , Adulto , Feminino , Humanos , Relações Interpessoais , MasculinoRESUMO
We report a case of pneumococcal pericarditis in a 13-year-old boy following allogeneic BMT from an HLA-identical unrelated donor. The post-transplant course was complicated by chronic GVHD which led to reinstitution of immunosuppressive therapy. Eight months after BMT the patient developed pericarditis with cardiac tamponade, and Streptococcus pneumoniae was isolated in the pericardiocentesis fluid. This is the first reported case of pneumococcal pericarditis after BMT. Although pericardial effusions after allogeneic BMT are often sterile and related to conditioning therapy or associated with chronic GVHD, rapid microbiological investigation and empirical treatment with antibiotics are necessary. Prophylaxis for pneumococcal infection in patients with chronic GVHD is recommended.
Assuntos
Transplante de Medula Óssea/efeitos adversos , Tamponamento Cardíaco/complicações , Doença Enxerto-Hospedeiro/complicações , Pericardite/etiologia , Infecções Pneumocócicas/etiologia , Adolescente , Humanos , Leucemia Mielogênica Crônica BCR-ABL Positiva/complicações , Leucemia Mielogênica Crônica BCR-ABL Positiva/terapia , Masculino , Pericardite/complicações , Infecções Pneumocócicas/complicaçõesRESUMO
PURPOSE: Using the data from the GETMON ("Grupo Español de Trasplante de Medula Osea en Niños") we carried out a retrospective analysis of the results of allogeneic bone marrow transplantation (alloBMT) compared to autologous bone marrow transplantation (ABMT) in 113 paediatric patients with acute lymphoblastic leukaemia (ALL) in second remission. Transplants were performed by the following centers, from April 1983 to December 1991: H. Vall d'Hebrón and H. Sant Pau from Barcelona, H. Ramón y Cajal and H. Niño Jesús from Madrid and H. Marqués de Valdecilla from Santander. PATIENTS AND METHODS: The study included 113 patients between the ages of two and 16 years with ALL in second remission at marrow transplant. Fifty-six underwent alloBMT and 57 ABMT. Both groups were homogeneous with respect to age, sex, immunophenotype, duration of first remission, risk at diagnosis, percentage of early and late relapses, percentage with marrow or extramedullary relapse prior to transplant, time interval from attainment of second remission to transplant, and conditioning regimens applied for marrow transplant, with predominance of chemoradiotherapy in both. RESULTS: Haematologic recovery was observed to be faster in alloBMT than in ABMT. A granulocyte count > 0.5 x 10(9)/l was reached in alloBMT patients in a median of 19 days and in ABMT patients in a median of 25 days (p < 0.001). Early procedure-related death after ABMT occurred only in one patient (1.75%) and was caused by hepatic veno-occlusive disease. In the alloBMT group, the incidence was 25%. GVHD and infection were the most common causes. Actuarial DFS for alloBMT was 38.8 +/- 6.7% at 8.5 years versus 29.2 +/- 6.5% at 4.5 years for ABMT, p = NS. No significant differences of actuarial DFS were found between alloBMT or ABMT in patients according to leukocyte count and risk at diagnosis, neither with first remission duration, nor with remission duration at transplant. A separate analysis of actuarial DFS for each group shows that in ABMT group DFS was significantly greater in patients who had presented a late relapse (> 30 months) 61.1 +/- 13.8%, than those who had presented an early relapse (< 30 months) 18.3 +/- 6.5% (p < 0.005). Probability of relapse was significantly greater in ABMT (70%) compared to alloBMT (46%) (p < 0.025). Transplant offers a better DFS in extramedullary relapses compared to isolated or combined bone marrow relapses: 71.4 +/- 17.1% with alloBMT and 38.1 +/- 14.7% with ABMT (p = NS). CONCLUSIONS: In our experience we observed a better DFS with alloBMT compared with ABMT, overcoat in early relapses, but without significant difference. A higher relapse rate in ABMT group is partially compensated by more early deaths in alloBMT offers a few survival possibilities in patients with medullary relapses whose first remission lasted less than 30 months.
Assuntos
Transplante de Medula Óssea/estatística & dados numéricos , Leucemia-Linfoma Linfoblástico de Células Precursoras/terapia , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Transplante de Medula Óssea/efeitos adversos , Transplante de Medula Óssea/mortalidade , Causas de Morte , Criança , Pré-Escolar , Terapia Combinada , Intervalo Livre de Doença , Feminino , Humanos , Imunofenotipagem , Tábuas de Vida , Masculino , Recidiva Local de Neoplasia , Células-Tronco Neoplásicas/imunologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/mortalidade , Leucemia-Linfoma Linfoblástico de Células Precursoras/patologia , Radioterapia/estatística & dados numéricos , Indução de Remissão , Estudos Retrospectivos , Espanha/epidemiologia , Análise de Sobrevida , Transplante Autólogo , Transplante Homólogo , Resultado do TratamentoRESUMO
A fatal case of meningoencephalitis caused by Scedosporium inflatum (Scedosporium prolificans) in a 5-year-old boy with acute myeloblastic leukemia who was given intrathecal treatment is reported. Itraconazole treatment was ineffective. The fungus was identified on brain sections at autopsy and was not observed in any other organ. As no other portal of entry was detected, meningoencephalitis may have originated via direct introduction of the fungus at therapeutic lumbar puncture.
Assuntos
Meningoencefalite/etiologia , Fungos Mitospóricos/isolamento & purificação , Micoses/etiologia , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Pré-Escolar , Evolução Fatal , Humanos , MasculinoRESUMO
We report a retrospective analysis on 46 pediatric patients (median age 9 years, range 1-17 years) with non-Hodgkin's lymphoma (NHL), transplanted in six Spanish centers. Fourteen patients underwent allogeneic bone marrow transplantation (BMT) and 32 autologous BMT. Most patients were boys (36 of 46). Twenty one cases were of lymphoblastic lymphoma, 19 Burkitt's lymphoma and six diffuse large cell lymphoma. Maximal Murphy's stage any time before BMT was stage III in 17 cases and stage IV in 29 cases. At BMT, 13 cases were in first CR, 21 in second CR, seven in third CR, four with sensitive active disease and one with refractory disease. All patients transplanted in CRl were considered candidates for BMT because of delayed CR (two cases), failure of the first-line therapy (seven cases) or central nervous system (CNS) or BM infiltration at diagnosis (four cases). Conditioning therapy included TBI in 33 patients and 13 cases were conditioned with chemotherapy alone. Toxic mortality was 13% (three of 14 in the allogeneic BMT group and three of 32 in the autologous group). No toxic deaths were registered in 13 patients undergoing BMT in CR1 (three allogeneic BMT and ten autologous BMT). Twelve patients relapsed 1-7 months after BMT. Overall event-free survival (EFS) was 58% (42-73%; confidence interval (CI) 95%), with a median follow-up of 33 months. EFS was similar for allogeneic BMT and autologous patients. Disease status at BMT was the only predictive factor for EFS (P < 0.01). There were no significant differences between patients in CR1 (82.5%) and CR2 (68%).(ABSTRACT TRUNCATED AT 250 WORDS)