Your browser doesn't support javascript.
loading
Mostrar: 20 | 50 | 100
Resultados 1 - 16 de 16
Filtrar
1.
Am J Hematol ; 2021 Feb 19.
Artigo em Inglês | MEDLINE | ID: mdl-33606297

RESUMO

Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for bone marrow failure or hematopoietic malignant diseases for Fanconi anemia (FA) patients. Although results have improved over the last decades, reaching more than 90% survival when a human leukocyte antigen (HLA)-identical donor is available, alternative HCT donors are still less reported. We compared HCT outcomes using HLA-mismatched unrelated donors (MMUD; n = 123) or haplo-identical donors (HDs), either using only in vivo T cell depletion (n = 33) or T cells depleted in vivo with some type of graft manipulation ex vivo (n = 59) performed for FA between 2000 and 2018. Overall survival (OS) by 24 months was 62% (53-71%) for MMUD, versus 80% (66-95%) for HDs with only in vivo T cell depletion and 60% (47-73%) for HDs with in vivo and ex vivo T cell depletion (p = .22). Event-free survival (EFS) was better for HD-transplanted FA patients with only in vivo T cell depletion 86% (73-99%) than for those transplanted from a MMUD 58% (48-68%) or those with graft manipulation 56% (42-69%) (p = .046). Grade II-IV acute graft-versus-host disease (GVHD) was 41% (MMUD) versus 40% (HDs with no graft manipulation) versus 17% (HDs with T cell depleted graft), (p = .005). No differences were found for the other transplant related outcomes. These data suggest that HDs might be considered as an alternative option for FA patients with better EFS using unmanipulated grafts.

2.
Pediatr Blood Cancer ; 67(9): e28233, 2020 09.
Artigo em Inglês | MEDLINE | ID: mdl-32614145

RESUMO

BACKGROUND: We conducted a national multicenter retrospective study in France to evaluate the efficacy and tolerance of ruxolitinib in children with steroid-refractory acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplant. PROCEDURE: Patients were recruited from the 15 pediatric transplantation centers. Transplanted patients were eligible if they met the following criteria: aged ≤ 18 years at transplantation, receiving a myeloablative allogeneic hematopoietic stem cell transplant, having an aGVHD of grade ≥2, and treated with ruxolitinib for steroid-refractory aGVHD. RESULTS: Twenty-nine patients received ruxolitinib for steroid-refractory aGVHD. Six patients achieved a complete response at day 28 after the start of treatment but finally 19 patients (65.5%) achieved a complete response (CR) with a median delay of 41 days (5-93 days). Two patients had a partial response. All patients who achieved CR or partial response discontinued corticosteroid treatment. Eight patients showed treatment failure. The overall response rate was 72.4%. Twenty-three of 29 patients were alive at a median follow-up of 685 days (177-1042 days) after the hematopoietic stem cell transplantation. Viral replication was observed in 41.4% of cases. We did not observe severe hematological adverse events and cytopenia requiring a modification of ruxolitinib doses always resolved. The median initial dose of ruxolitinib was 12.6 mg/m2 /day with an important range. We could not demonstrate any relationship between initial dose and effectiveness. CONCLUSION: Ruxolitinib may constitute a promising second-line treatment for children with steroid-refractory aGVHD that should be validated in a prospective large-scale pharmacokinetic and efficacy trial.

3.
Pediatr Blood Cancer ; 67(6): e28305, 2020 06.
Artigo em Inglês | MEDLINE | ID: mdl-32307866

RESUMO

CONTEXT: Acute myeloid leukemia (AML) is a rare disease in children, with only 50% to 60% event-free survival. Among patients with AML, 10% do not respond to first-line chemotherapy. There is no recommendation concerning second-line treatments. Gemtuzumab ozogamicin (GO) is a monoclonal antibody targeting CD33, linked to calicheamicin. We report the efficacy and tolerance of a salvage regimen of fludarabin, cytarabine, and GO (FLA-GO) in patients refractory to first-line treatment. METHODS: Eight patients (median age 14.5 years), who had more than 2% minimal residual disease (MRD) by flow cytometry (MRD flow), received gemtuzumab 3 mg/m² on days 1, 4, 7, associated with cytarabine 2000 mg/m² and fludarabin 30 mg/m² on days 1 to 5. RESULTS: Six patients achieved complete remission (CR) (blast count morphology ≤5 × 10-2 , CR-MRD flow <1 × 10-3 for four patients). Five patients received a second course. We observed 11 episodes of febrile neutropenia, including 6 septicemias without complication. There was no fungal infection or toxic death. Two patients received granulocyte colony stimulating factor. One patient had partial platelet recovery; one, prolonged pancytopenia. All patients received hematopoietic stem cell transplantation (HSCT). We observed five mild-to-severe sinusoidal obstruction syndromes during HSCT procedures, particularly in patients who did not receive defibrotide prophylaxis. At the date of last contact (median follow-up: 58 months; range: 22-78), six patients were in continuous CR with negative MRD. Two patients died of post-HSCT relapse. CONCLUSION: FLA-GO is a good salvage regimen for pediatric refractory AML, with significant but acceptable toxicity. HSCT is mandatory to achieve sustained CR in these patients.


Assuntos
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Resistencia a Medicamentos Antineoplásicos , Transplante de Células-Tronco Hematopoéticas/mortalidade , Leucemia Mieloide Aguda/terapia , Recidiva Local de Neoplasia/terapia , Terapia de Salvação , Adolescente , Criança , Terapia Combinada , Citarabina/administração & dosagem , Feminino , Seguimentos , Gemtuzumab/administração & dosagem , Humanos , Leucemia Mieloide Aguda/patologia , Masculino , Recidiva Local de Neoplasia/patologia , Prognóstico , Estudos Retrospectivos , Taxa de Sobrevida , Vidarabina/administração & dosagem , Vidarabina/análogos & derivados
4.
Biol Blood Marrow Transplant ; 26(12): 2285-2291, 2020 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-32007639

RESUMO

Graft-versus-host disease (GVHD) is an important challenge and a major cause of morbidity and mortality in children after hematopoietic stem cell transplant (HSCT). Herein we report our institution's experience of goal-oriented Bayesian monitoring for cyclosporine (CsA) used alone as GVHD prophylaxis during the post-transplant period in pediatric patients with thalassemia major (TM) or sickle cell anemia (SCA) undergoing HLA-matched HSCT. We also studied evolution of chimerism. Twenty-six consecutive patients (SCA, 14; TM, 12) underwent matched sibling donor (MSD) HSCT from 2004 to 2014. All patients received a myeloablative conditioning regimen. GVHD prophylaxis consisted of 20 mg/kg antithymocyte globulin in the conditioning regimens and then CsA alone in the post-transplant period. Target CsA trough blood concentration (TBC) was 150 ± 20 ng/mL. At last follow-up, all patients were alive and free of disease, even in cases of mixed chimerism. Engraftment occurred in all patients. No patient developed grades II to IV acute GVHD, 4 patients developed acute grade I skin GVHD, and only 1 presented with chronic pulmonary GVHD. A better control of GVHD and immunosuppression by a strict monitoring of CsA TBC as described herein is promising and could play a crucial role. Further investigations are required, but this study opens new perspectives to improve survival and safety of HSCT from alternative donors in TM and SCA to levels compatible with that obtained with MSDs.

5.
Eur J Oncol Nurs ; 38: 1-7, 2019 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-30717930

RESUMO

PURPOSE: Immunosuppressive therapy following pediatric hematopoietic stem cell transplantation is essential for the patient's prognosis, as the antibioprophylaxis and the isolation measures. But medication adherence is suboptimal for children and adolescents, from 52 to 73% in literature. The aim of this study is to provide an understanding of medication adherence after pediatric allogeneic stem cell transplantation (SCT), by identifying facilitators and barriers. METHOD: Semi-structured interviews were conducted by a pharmacist with caregivers and healthcare providers in a pediatric centre. Four topics were discussed: transplantation, post-transplantation therapies, caregivers' experience and the healthcare system. Interviews were audiotaped, transcribed and analysed by inductive approach. FINDINGS: Semi-structured interviews with 15 caregivers and 21 healthcare providers identified factors of medication adherence and hygiene measures. The long-term nature of therapy and difficult transitions of care were identified as major barriers. Recognizing the benefits of medication and parental involvement are facilitators. Furthermore, caregivers expressed the need to take into consideration the family entity. They would like also to receive earlier information from healthcare providers before hospital discharge. Those needs were not always identified by healthcare providers. CONCLUSION: This analysis revealed barriers and facilitators to the medication adherence and to the care. It demonstrated similarities and differences between caregivers and healthcare providers' perceptions and has thereby initiated an improvement process of the healthcare system. As part of this process, medical and paramedical healthcare providers at this French pediatric centre are currently working on a support program for post-alloSCT hospital-home transition.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Adesão à Medicação , Adolescente , Fatores Etários , Atitude do Pessoal de Saúde , Cuidadores , Criança , Feminino , Pessoal de Saúde , Humanos , Imunossupressores/uso terapêutico , Masculino , Pais , Alta do Paciente , Pesquisa Qualitativa
6.
Biol Blood Marrow Transplant ; 25(4): 734-742, 2019 04.
Artigo em Inglês | MEDLINE | ID: mdl-30385256

RESUMO

We previously reported in a French prospective randomized study that transplantation of 2 unrelated cord blood (UCB) units instead of 1 unit does not decrease the risk of transplantation failure but may enhance alloreactivity. Here we evaluated the influence of pretransplantation minimal residual disease (MRD) on leukemia relapse and survival after single- versus double-UCB transplantation (UCBT). Among 137 children and young adults who underwent UCBT in this randomized study, 115 had available data on MRD assessment done immediately before initiation of the pretransplantation conditioning regimen. MRD was considered positive at a level of ≥10-4, which was the case of 43 out of 115 patients. Overall, the mean 3-year survival probability was 69.1 ± 4.4%, and it was not significantly influenced by the MRD level: 70.7 ± 5.4% in MRD-negative (<10-4) patients (n = 72), 71.1 ± 9.4% in MRD-positive patients with 10-4 ≤ MRD <10-3 (n = 26) and 58.8 ± 11.9% in MRD-positive patients with ≥10-3 (n = 17). In the MRD-positive group, the mean risk of relapse was significantly lower in the double-UCBT arm compared with the single-UCBT arm (10.5 ± 7.2% versus 41.7 ± 10.4%; P = .025) leading to a higher mean 3-year survival rate (82.6 ± 9.3% versus 53.6 ± 10.3%; P = .031). This difference was observed only in patients who had not received antithymocyte globulin during their conditioning regimen. In the MRD-negative group, there was no differencebetween the single- and the double-UCBT arms. We conclude that even in cases of positive pretransplantation MRD, UCBT in children and young adults with acute leukemia yields a high cure rate, and that a double-unit strategy may enhance the graft-versus-leukemia effect and survival in these patients.


Assuntos
Transplante de Células-Tronco de Sangue do Cordão Umbilical/métodos , Leucemia Mieloide Aguda/terapia , Adulto , Feminino , Humanos , Masculino , Estudos Prospectivos , Adulto Jovem
7.
Antiviral Res ; 160: 1-9, 2018 12.
Artigo em Inglês | MEDLINE | ID: mdl-30315875

RESUMO

INTRODUCTION: A child with severe combined immunodeficiency (SCID) had an influenza A(H1N1)pdm09 infection with viral excretion longer than 6 months, during 2013-2014 influenza season, despite cord blood transplantation and antiviral treatments. METHODS: Conventional real-time RT-PCR methods were used to estimate viral load and to detect the presence of the common N1 neuraminidase (NA) H275Y substitution responsible for oseltamivir resistance. Next-generation sequencing (NGS) of influenza viruses was performed retrospectively to characterize viral quasispecies in specimens. RESULTS: The patient was first treated with oral oseltamivir, leading to detection of low-levels of NA-H275Y substitution. Concomitant cord blood cell transplantation, intravenous administration of zanamivir and immunoglobulins led to an increase in white blood cells and influenza viral load decrease. A viral rebound occurred as soon as the antiviral treatment was discontinued. Eventually, influenza viral load was negated with immune reconstitution. NGS found influenza quasispecies harboring NA-E119A substitution (10.3%). Moreover, NGS showed that viral genomic diversity evolved under antiviral treatment and immune status. CONCLUSIONS: Conventional virological techniques were sufficient for influenza infection follow-up but NGS performances allowed characterization of viral variants evolution in this specific case of prolonged influenza virus infection. New and efficient treatments against influenza in immunocompromised patients are needed.


Assuntos
Variação Genética , Vírus da Influenza A Subtipo H1N1/genética , Vírus da Influenza A Subtipo H1N1/isolamento & purificação , Influenza Humana/patologia , Influenza Humana/virologia , Imunodeficiência Combinada Severa/complicações , Antivirais/administração & dosagem , Antivirais/farmacologia , Criança , Transplante de Células-Tronco de Sangue do Cordão Umbilical , Farmacorresistência Viral , Humanos , Imunoglobulinas Intravenosas/administração & dosagem , Vírus da Influenza A Subtipo H1N1/classificação , Vírus da Influenza A Subtipo H1N1/efeitos dos fármacos , Influenza Humana/tratamento farmacológico , Mutação de Sentido Incorreto , Neuraminidase/genética , Oseltamivir/administração & dosagem , Oseltamivir/farmacologia , Reação em Cadeia da Polimerase em Tempo Real , Carga Viral , Proteínas Virais/genética , Zanamivir/administração & dosagem
8.
J Pediatr ; 198: 46-52, 2018 07.
Artigo em Inglês | MEDLINE | ID: mdl-29709343

RESUMO

OBJECTIVE: To study the risk of catheter-associated thrombosis (CAT) between peripherally inserted central catheters (PICCs) and tunneled central venous catheters in children with leukemia. STUDY DESIGN: We analyzed all PICCs and conventional tunneled catheters placed in patients aged <18 years and admitted to our institute for leukemia treatment between February 2008 and April 2014. Cases of symptomatic CAT were confirmed by ultrasound and treated with low-molecular-weight heparin. RESULTS: During the study period, 157 PICCs and 138 conventional tunneled catheters were placed in 192 patients with leukemia. CAT incidence was 1.5% (n = 2) in the conventional tunneled catheter group and 10.2% (n = 16) in the PICC group. The OR for CAT occurrence after PICC vs conventional tunneled catheter placement was 5.6 (95% CI, 1.2-26.5). CONCLUSION: Our results suggest that the use of PICCs in children with leukemia increases the risk of CAT in comparison with the use of conventional tunneled catheters. Further randomized controlled studies are needed to characterize this risk and to better define indications.


Assuntos
Cateterismo Venoso Central/efeitos adversos , Cateterismo Periférico/efeitos adversos , Cateterismo Periférico/instrumentação , Cateteres Venosos Centrais/efeitos adversos , Leucemia/terapia , Trombose Venosa/epidemiologia , Criança , Feminino , Humanos , Incidência , Masculino , Estudos Retrospectivos , Fatores de Risco
9.
Fundam Clin Pharmacol ; 32(3): 323-329, 2018 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-29315849

RESUMO

Continuous intravenous (IV) infusion has been shown to be the best option to administer vancomycin because of its time-dependent bactericidal activity. Available IV vancomycin dosing guidelines in pediatrics with normal renal function leads to less than 50% of patients achieving a vancomycin serum concentration (Css) in the target range (15-20 mg/L). The primary objective of this study was to prospectively validate an age-based dosing regimen in pediatric oncology-hematology. The secondary objective was to investigate the influence on Css attainment of different variables. A continuous IV dosing nomogram was built by retrospective study (2000-2010) on Bayesian dosing adjustments performed in 161 patients. This study assessed the prospective validation of this age-based nomogram and the influence on Css attainment of variables as the gender, underlying disease (oncology or hematology), and hematopoietic stem cell transplantation (HSCT) before receiving vancomycin therapy. A total of 94 patients aged from 4.3 months to 17.9 years old with normal renal function were eligible for the prospective validation. Fifty-five of those patients (58.5%) achieved the target range of vancomycin Css. There was no significant difference between age groups (P = 0.816) and no influence of gender (P = 0.500). There was a nonsignificant trend to a better target attainment in oncology patients (69.2% vs. hematology 54.4%, P = 0.142) and patients who did not undergo HSCT (63.3% vs. 33.3%, P = 0.031). This study proposed an age-based nomogram prospectively validated which near 60% of patients of each age class achieving the target range of Css.


Assuntos
Antibacterianos/administração & dosagem , Técnicas de Apoio para a Decisão , Cálculos da Dosagem de Medicamento , Hematologia , Hospitalização , Oncologia , Vancomicina/administração & dosagem , Adolescente , Fatores Etários , Antibacterianos/efeitos adversos , Antibacterianos/sangue , Antibacterianos/farmacocinética , Criança , Pré-Escolar , Monitoramento de Medicamentos , Feminino , Humanos , Lactente , Infusões Intravenosas , Masculino , Nomogramas , Estudos Prospectivos , Reprodutibilidade dos Testes , Vancomicina/efeitos adversos , Vancomicina/sangue , Vancomicina/farmacocinética
10.
Bone Marrow Transplant ; 53(4): 400-409, 2018 04.
Artigo em Inglês | MEDLINE | ID: mdl-29330405

RESUMO

The question of the best donor type between haploidentical (HAPLO) and matched-related donors (MRD) for patients with advanced HL receiving an allogeneic hematopoietic cell transplantation (allo-HCT) is still debated. Given the lack of data comparing these two types of donor in the setting of non-myeloablative (NMA) or reduced-intensity (RIC) allo-HCT, we performed a multicentre retrospective study using graft-vs.-host disease-free relapse-free survival (GRFS) as our primary endpoint. We analysed the data of 151 consecutive HL patients who underwent NMA or RIC allo-HCT from a HAPLO (N = 61) or MRD (N = 90) between January 2011 and January 2016. GRFS was defined as the probability of being alive without evidence of relapse, grade 3-4 acute GVHD or chronic GVHD. In multivariable analysis, MRD donors were independently associated with lower GRFS compared to HAPLO donors (HR = 2.95, P < 0.001). Disease status at transplant other than CR was also associated with lower GRFS in multivariable analysis (HR = 1.74, P = 0.01). In addition, the administration of ATG was independently linked to higher GRFS (HR = 0.52, P = 0.009). In summary, we observed significantly higher GRFS in HL patients receiving an allo-HCT using the HAPLO PT-Cy platform compared to MRD.


Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Doença de Hodgkin/terapia , Doadores de Tecidos , Transplante Haploidêntico/métodos , Adolescente , Adulto , Idoso , Criança , Intervalo Livre de Doença , Feminino , Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Doença de Hodgkin/complicações , Doença de Hodgkin/mortalidade , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Retrospectivos , Transplante Homólogo , Adulto Jovem
12.
Pediatr Blood Cancer ; 64(8)2017 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-28097791

RESUMO

BACKGROUND: Sickle cell anaemia (SCA) is a severe hereditary haemoglobinopathy characterised by haemorheological abnormalities, which play a role in the occurrence of several acute and chronic clinical complications. While ßS -haplotypes and alpha-thalassaemia modulate SCA clinical severity, their effects on blood rheology have been incompletely described. The aim of this study was to test the effects of these genetic modifiers on the haemorheological properties and clinical complication of children with SCA. PROCEDURE: Steady-state haemorheological profile, biological parameters, ßS -haplotypes, alpha-globin status, vaso-occlusive crisis (VOC) and acute chest syndrome frequencies were analysed in 128 children (aged 5 to 18 years) with SCA. RESULTS: Patients with alpha-thalassaemia showed increased red blood cell (RBC) deformability and aggregation compared to those without. Median VOC rate was higher in patients with homozygous alpha-thalassaemia compared to those with a normal alpha genotype. Conversely, the haemorheological profile and clinical complications were not influenced by the ßS -haplotypes in our study. CONCLUSION: Our results demonstrate that alpha-thalassaemia is associated with higher risk for VOC events in children with SCA, which may be due in part to its effects on RBC deformability and aggregation.


Assuntos
Anemia Falciforme/complicações , Eritrócitos/patologia , Talassemia alfa/complicações , Síndrome Torácica Aguda/patologia , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Masculino , Reologia
13.
Eur J Haematol ; 98(3): 296-301, 2017 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-27981643

RESUMO

OBJECTIVES: Because of the increased hemolytic rate, a significant proportion of patients with sickle cell disease (SCD) are prone to develop cholelithiasis. The present study investigated the role of several genetic factors (UGT1A1 promoter (TA)n repeat polymorphism, alpha-globin status), hematological parameters, clinical severity, and hydroxyurea (HU) therapy on the occurrence of cholelithiasis in SCD. METHODS: One hundred and fifty-eight children (2-18 yr old) regularly followed at the University Hospital of Lyon (France) were included. A multivariate Cox model was used to test the associations between cholelithiasis and the different parameters analyzed. RESULTS: We confirmed that alpha-thalassemia and low basal reticulocyte (RET) count were independent protective factors for cholelithiasis while 7/7, 8/8 and 7/8 UGT1A1 (TA)n genotypes were independent predisposing factors for this complication. We also showed for the first time that HU treatment decreased the risk for cholelithiasis while frequent vaso-occlusive crises and acute chest syndrome events increased that risk. CONCLUSIONS: Our findings demonstrate that UGT1A1 (TA)n polymorphism is not the only factor triggering gallstone formation in SCD. Cholelithiasis is also modulated by RET count, the number of deleted alpha-genes, HU therapy and the frequency of vaso-occlusive events.


Assuntos
Alelos , Anemia Falciforme/complicações , Anemia Falciforme/genética , Colelitíase/etiologia , Genótipo , Glucuronosiltransferase/genética , Adolescente , Biomarcadores , Criança , Pré-Escolar , Colelitíase/diagnóstico , Colelitíase/epidemiologia , Feminino , Humanos , Incidência , Leucócitos , Masculino , Estudos Retrospectivos , Fatores de Risco , alfa-Globinas/genética
14.
Blood ; 127(26): 3450-7, 2016 06 30.
Artigo em Inglês | MEDLINE | ID: mdl-27099151

RESUMO

Transplantation of 2 unrelated cord blood (UCB) units instead of 1 has been proposed to increase the cell dose. We report a prospective randomized study, designed to compare single- vs double-UCB transplantation in children and young adults with acute leukemia in remission or myelodysplasia. Eligible patients had at least two 4-6 HLA-identical UCBs with >3 × 10(7) nucleated cells/kg for the first and >1.5 × 10(7) for the second. The primary end point was the 2-year cumulative incidence of transplantation strategy failure, a composite end point including transplant-related mortality (TRM), engraftment failure, and autologous recovery. Randomized patients who did not proceed to transplantation due to refractory disease were considered transplantation failures. A total of 151 patients were randomized and included in the intent-to-treat analysis; 137 were transplanted. Double-UCB transplantation did not decrease transplantation strategy failure (23.4% ± 4.9% vs 14.9% ± 4.2%). Two-year posttransplant survival, disease-free survival, and TRM were 68.8% ± 6.0%, 67.6% ± 6.0%, and 5.9% ± 2.9% after single-unit transplantation compared with 74.8% ± 5.5%, 68.1% ± 6.0%, and 11.6% ± 3.9% after double-unit transplantation. The final relapse risk did not significantly differ, but relapses were delayed after double-unit transplantation. Overall incidences of graft-versus-host disease (GVHD) were similar, but chronic GVHD was more frequently extensive after double-UCB transplantation (31.9% ± 5.7% vs 14.7% ± 4.3%, P = .02). In an exploratory subgroup analysis, we found a significantly lower relapse risk after double-unit transplantation in patients receiving total body irradiation without antithymocyte globulin (ATG), whereas the relapse risk was similar in the group treated with busulfan, cyclophosphamide, and ATG. Single-UCB transplantation with adequate cell dose remains the standard of care and leads to low TRM. Double-unit transplantation should be reserved for patients who lack such units. This trial was registered at www.clinicaltrials.gov as #NCT01067300.


Assuntos
Transplante de Células-Tronco de Sangue do Cordão Umbilical , Leucemia/terapia , Síndromes Mielodisplásicas/terapia , Condicionamento Pré-Transplante/métodos , Doença Aguda , Adolescente , Adulto , Soro Antilinfocitário/administração & dosagem , Criança , Pré-Escolar , Doença Crônica , Intervalo Livre de Doença , Feminino , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/mortalidade , Humanos , Leucemia/mortalidade , Masculino , Síndromes Mielodisplásicas/mortalidade , Taxa de Sobrevida , Irradiação Corporal Total , Adulto Jovem
15.
Eur J Haematol ; 96(4): 404-8, 2016 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-26072930

RESUMO

The aim of this study was to test the association between hematological/genetic factors and cerebral vasculopathy in children with sickle cell anemia (SCA). A group with cerebral vasculopathy (VASC) was composed of children who had stroke (n = 6), silent infarct (n = 11), or an abnormal transcranial Doppler (n = 5). Eighty-four patients had neither positive history of stroke or silent infarct, nor abnormal transcranial Doppler (NORM group). An intermediate group (COND; n = 15) was composed of SCA children with a conditional transcranial Doppler. Biological analyses were performed on samples obtained at steady state and before the beginning of any chronic treatment. The comparisons of the three groups demonstrated a protective effect of α-thalassemia against cerebral vasculopathy through its effects on hemoglobin and reticulocyte levels. Moreover, we observed higher frequency of G6PD deficiency in the VASC group compared with the other groups. Our study confirms the key role of α-thalassemia and G6PD status in the pathophysiology of cerebral vasculopathy in SCA children.


Assuntos
Anemia Falciforme/diagnóstico , Transtornos Cerebrovasculares/diagnóstico , Deficiência de Glucosefosfato Desidrogenase/diagnóstico , Talassemia alfa/diagnóstico , Anemia Falciforme/sangue , Anemia Falciforme/complicações , Anemia Falciforme/patologia , Transtornos Cerebrovasculares/sangue , Transtornos Cerebrovasculares/complicações , Transtornos Cerebrovasculares/patologia , Criança , Pré-Escolar , Feminino , Deficiência de Glucosefosfato Desidrogenase/sangue , Deficiência de Glucosefosfato Desidrogenase/complicações , Deficiência de Glucosefosfato Desidrogenase/patologia , Hemoglobinas/metabolismo , Humanos , Masculino , Contagem de Reticulócitos , Reticulócitos/patologia , Fatores de Risco , Ultrassonografia Doppler Transcraniana , Talassemia alfa/sangue , Talassemia alfa/patologia
16.
Br J Haematol ; 152(3): 322-30, 2011 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-21133884

RESUMO

We report the post-transplant lymphocyte subset recovery of 226 children treated with Unrelated Cord Blood transplant (UCBT) (n = 112) or Unrelated Bone Marrow Transplant (UBMT) (n = 114) for malignant or non-malignant diseases. Absolute numbers of natural killer (NK), B and T cells were monitored by flow cytometry up to 5 years post-transplant. Immunological endpoints were: time to achieve a CD3(+) cell count > 0·5 and 1·5 × 109/l, CD4(+) > 0·2 and 0·5 × 109/l, CD8(+) > 0·25 ×109/l, CD19(+) > 0·2 × 109/l, NK > 0·1 × 109/l. These endpoints were analysed through the use of cumulative incidence curves in the context of competing risks. CD8(+) T cell recovery was delayed after UCBT with a median time to reach CD8(+) T cells > 0·25 × 109/l of 7·7 months whereas it was 2·8 months in UBMT (P < 0·001). B cell recovery was better in UCBT, with a median time to reach CD19(+) cells > 0·2 × 109/l of 3·2 months in UCBT and 6·4 months in UBMT (P = 0·03). Median time for CD4(+) T cell and NK cell recovery was similar in UCBT and UBMT. CD4(+) T cells recovery was negatively correlated to age (better reconstitution in younger patients, P = 0·002). CD8(+) T cells recovery was shorter in recipients with a positive cytomegalovirus serology (P =0·001).


Assuntos
Transplante de Medula Óssea/métodos , Transplante de Células-Tronco de Sangue do Cordão Umbilical/métodos , Doenças Hematológicas/terapia , Subpopulações de Linfócitos/imunologia , Adolescente , Linfócitos B/imunologia , Criança , Pré-Escolar , Feminino , Doenças Hematológicas/imunologia , Humanos , Lactente , Células Matadoras Naturais/imunologia , Contagem de Linfócitos , Masculino , Infecções Oportunistas/imunologia , Subpopulações de Linfócitos T/imunologia , Resultado do Tratamento
SELEÇÃO DE REFERÊNCIAS
DETALHE DA PESQUISA
...