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1.
Pediatr Transplant ; 24(1): e13641, 2020 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-31880407

RESUMO

Myeloid neoplasms in children with sickle cell disease have been rarely reported, and the exact underlying connection between these two conditions is not clearly understood. Whether the acute myeloid leukemia in hydroxyurea-treated sickle cell patients is co-incidental or related to therapy remains an unanswered question. Herein, we report a 14-year-old girl of Haitian descent with sickle beta zero thalassemia on chronic hydroxyurea therapy who developed FMS-like tyrosine kinase 3 (FLT3)-mutated acute myeloid leukemia and underwent a complete disease remission following a combination chemotherapy with sorafenib and was subsequently treated using a T cell replete unmanipulated haploidentical bone marrow transplantation followed by post-transplant cyclophosphamide.

2.
J Clin Immunol ; 39(7): 653-667, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31376032

RESUMO

INTRODUCTION: Inflammatory bowel disease (IBD) affects approximately 1/3 of patients with chronic granulomatous disease (CGD). Comprehensive investigation of the effect of allogeneic hematopoietic cell transplantation (HCT) on CGD IBD and the impact of IBD on transplant outcomes is lacking. METHODS: We collected data retrospectively from 145 patients with CGD who had received allogeneic HCT at 26 Primary Immune Deficiency Treatment Consortium (PIDTC) centers between January 1, 2005 and June 30, 2016. RESULTS: Forty-nine CGD patients with IBD and 96 patients without IBD underwent allogeneic HCT. Eighty-nine percent of patients with IBD and 93% of patients without IBD engrafted (p = 0.476). Upper gastrointestinal acute GVHD occurred in 8.5% of patients with IBD and 3.5% of patients without IBD (p = 0.246). Lower gastrointestinal acute GVHD occurred in 10.6% of patients with IBD and 11.8% of patients without IBD (p = 0.845). The cumulative incidence of acute GVHD grades II-IV was 30% (CI 17-43%) in patients with IBD and 20% (CI 12-29%) in patients without IBD (p = 0.09). Five-year overall survival was equivalent for patients with and without IBD: 80% [CI 66-89%] and 83% [CI 72-90%], respectively (p = 0.689). All 33 surviving evaluable patients with a history of IBD experienced resolution of IBD by 2 years following allogeneic HCT. CONCLUSIONS: In this cohort, allogeneic HCT was curative for CGD-associated IBD. IBD should not contraindicate HCT, as it does not lead to an increased risk of mortality. This study is registered at clinicaltrials.gov NCT02082353.

3.
Leuk Lymphoma ; 60(2): 385-394, 2019 02.
Artigo em Inglês | MEDLINE | ID: mdl-29966479

RESUMO

The purpose was to describe the incidence and risk factors of congestive heart failure (CHF) among children with acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). We included 2053 children (≤18 years) with first primary ALL and AML diagnosed 1992-2010 and registered in the Pediatric Oncology Group of Ontario Networked Information System. We identified CHF events through linked administrative databases. At 10 years, the cumulative incidence of CHF was 1.7% in ALL and 7.5% in AML. Factors associated with CHF in ALL were female gender, age <1 year at cancer diagnosis, irradiation and cumulative anthracycline dose ≥250 mg/m2. Irradiation was the only risk factor in AML patients. Of the 23 patients with CHF during active therapy, one developed CHF following treatment completion. Incidence of CHF were 1.7% in ALL and 7.5% in AML. Most with CHF during active therapy did not develop CHF after treatment completion.

4.
Cancer ; 125(6): 963-971, 2019 03 15.
Artigo em Inglês | MEDLINE | ID: mdl-30521100

RESUMO

BACKGROUND: Langerhans cell histiocytosis (LCH) is a rare myeloid neoplasm characterized by the presence of abnormal CD1a-positive (CD1a+ )/CD207+ histiocytes. Hemophagocytic lymphohistiocytosis (HLH) represents a spectrum of hyperinflammatory syndromes typified by the dysregulated activation of the innate and adaptive immune systems. Patients with LCH, particularly those with multisystem (MS) involvement, can develop severe hyperinflammation mimicking that observed in HLH. Nevertheless, to the authors' knowledge, little is known regarding the prevalence, timing, risk factors for development, and outcomes of children and young adults who develop HLH within the context of MS-LCH (hereafter referred to LCH-associated HLH). METHODS: To gain further insights, the authors conducted a retrospective, multicenter study and collected data regarding all patients diagnosed with MS-LCH between 2000 and 2015. RESULTS: Of 384 patients with MS-LCH, 32 were reported by their primary providers to have met the diagnostic criteria for HLH, yielding an estimated 2-year cumulative incidence of 9.3% ± 1.6%. The majority of patients developed HLH at or after the diagnosis of MS-LCH, and nearly one-third (31%) had evidence of an intercurrent infection. Patient age <2 years at the time of diagnosis of LCH; female sex; LCH involvement of the liver, spleen, and hematopoietic system; and a lack of bone involvement each were found to be independently associated with an increased risk of LCH-associated HLH. Patients with MS-LCH who met the criteria for HLH had significantly poorer 5-year survival compared with patients with MS-LCH who did not meet the criteria for HLH (69% vs 97%; P < .0001). CONCLUSIONS: Given its inferior prognosis, further efforts are warranted to enhance the recognition and optimize the treatment of patients with LCH-associated HLH.


Assuntos
Sistema Hematopoético/imunologia , Histiocitose de Células de Langerhans/complicações , Fígado/imunologia , Linfo-Histiocitose Hemofagocítica/epidemiologia , Baço/imunologia , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Sistema Hematopoético/patologia , Histiocitose de Células de Langerhans/imunologia , Humanos , Lactente , Recém-Nascido , Fígado/patologia , Linfo-Histiocitose Hemofagocítica/imunologia , Masculino , Prognóstico , Estudos Retrospectivos , Baço/patologia , Adulto Jovem
5.
Mediterr J Hematol Infect Dis ; 8(1): e2016033, 2016.
Artigo em Inglês | MEDLINE | ID: mdl-27413525

RESUMO

BACKGROUND: Bone is the most common organ of involvement in patients with Langerhans cell histiocytosis (LCH), which is often painful and associated with significant morbidity from pathological fractures. Current first-line treatments include chemotherapy and steroids that are effective but often associated with adverse effects, whereas the disease may reactivate despite an initial response to first-line agents. Bisphosphonates are osteoclast inhibitors that have shown to be helpful in treating bone lesions of LCH. To date, there are no large international studies to describe their role in treating bone lesions of LCH. METHOD: We conducted a multicenter retrospective review of 13 patients with histologically proven LCH, who had received bisphosphonates either at diagnosis or at disease reactivation. RESULTS: Ten patients (77%) had a single system bone disease, and 3 (23%) had bone lesions as part of multisystem disease. Median follow-up time post-bisphosphonate therapy was 4.6 years (range, 0.8 to 8.2 years). Treatment with bisphosphonates was associated with significant pain relief in almost all patients. Twelve (92%) achieved resolution of active bone lesions, and 10 out of them had no active disease for a median of 3.5 years (range, 0.8 to 5 years). One patient did not respond. No major adverse effects were reported in this series. CONCLUSION: Bisphosphonates are well-tolerated drugs that can significantly improve bone pain and induce remission in active bone LCH. Future prospective studies evaluating the role of bisphosphonates in LCH are warranted.

6.
Pediatr Blood Cancer ; 63(2): 355-7, 2016 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-26398727

RESUMO

Hematopoietic stem cell transplantation (HSCT) is currently the only available curative therapy for X-linked inhibitor of apoptosis (XIAP) deficiency. Myeloablative conditioning regimens are associated with high mortality rates. Reduced-intensity conditioning (RIC) is recommended in order to decrease treatment-related toxicities, but RIC regimens increase the risk for mixed donor-recipient chimerism that may progress to graft loss. We report our experience with a patient with XIAP deficiency who was successfully treated with allogeneic HSCT using a RIC protocol. Post-transplant chimerism was vigilantly monitored and maintained with donor lymphocyte infusions and a stem cell boost to a level that prevented hemophagocytic lymphohistiocytosis recurrence.


Assuntos
Doenças Genéticas Ligadas ao Cromossomo X/cirurgia , Transplante de Células-Tronco Hematopoéticas/métodos , Transtornos Linfoproliferativos/cirurgia , Agonistas Mieloablativos/uso terapêutico , Condicionamento Pré-Transplante/métodos , Alemtuzumab , Anticorpos Monoclonais Humanizados/administração & dosagem , Pré-Escolar , Humanos , Masculino , Melfalan/administração & dosagem , Transplante Homólogo/métodos , Vidarabina/administração & dosagem , Vidarabina/análogos & derivados
7.
Pediatr Blood Cancer ; 62(12): 2162-6, 2015 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-26179251

RESUMO

BACKGROUND: Children with Langerhans cell histiocytosis (LCH) and single-bone CNS-risk lesions have been reported to be at increased risk of diabetes insipidus (DI), central nervous system neurodegeneration (CNS-ND), and recurrence of disease. However, it is unknown whether the addition of chemotherapy or radiotherapy changes outcomes in these patients. METHODS: Ten pediatric institutions across North America and Europe contributed data of their patients with LCH and single-bone CNS-risk lesions. Clinical information on age, sex, specific craniofacial site involvement, and intracranial extension at diagnosis, therapy, and disease course was collected for all eligible patients. RESULTS: The final analysis included 93 eligible children who were either treated with systemic therapy (chemotherapy, chemo-radiotherapy, or radiotherapy) or local therapy (biopsy, curettage, and/or intralesional steroids). Fifty-nine patients had systemic and 34 had local therapy. The 5-year event-free survival (EFS) and overall survival (OS) were 80 ± 5% and 98 ± 2% in the systemic therapy group versus 85 ± 6% and 95 ± 5% in the local therapy group. There was no statistically significant difference between either group with regard to EFS (P = 0.26) and OS (P = 0.78). On multivariable analysis, there was no significant difference among the two treatment groups after adjusting for site and intracranial soft tissue extension, nor any trend favoring systemic therapy (HR = 2.26, 95% CI = 0.77-6.70; P = 0.14). CONCLUSION: Systemic therapy may not reduce the risk of recurrence or late sequelae in children with LCH and single-bone CNS-risk lesions as compared to local treatment.


Assuntos
Doenças Ósseas/mortalidade , Doenças Ósseas/terapia , Histiocitose de Células de Langerhans/mortalidade , Histiocitose de Células de Langerhans/terapia , Doenças Neurodegenerativas/mortalidade , Doenças Neurodegenerativas/terapia , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Seguimentos , Humanos , Lactente , Masculino , Estudos Retrospectivos , Taxa de Sobrevida
8.
J Clin Oncol ; 33(5): 501-9, 2015 Feb 10.
Artigo em Inglês | MEDLINE | ID: mdl-25559816

RESUMO

PURPOSE: The objective of this study was to describe the diagnostic yield and complication rate of bronchoalveolar lavage (BAL) and lung biopsy in the evaluation of pulmonary lesions in patients with cancer and recipients of hematopoietic stem-cell transplantation (HSCT). METHODS: We conducted a systematic literature review and performed electronic searches of Ovid MEDLINE, EMBASE, and Cochrane Central Register of Controlled Trials. Studies were included if patients had cancer or were recipients of HSCT, and if they underwent BAL or lung biopsy for the evaluation of pulmonary lesions. Only English language publications were included. RESULTS: In all, 14,148 studies were screened; 72 studies of BAL and 31 of lung biopsy were included. The proportion of procedures leading to any diagnosis was similar by procedure type (0.53 v 0.54; P = .94) but an infectious diagnosis was more common with BAL compared with lung biopsy (0.49 v 0.34; P < .001). Lung biopsy more commonly led to a noninfectious diagnosis (0.43 v 0.07; P < .001) and was more likely to change how the patient was managed (0.48 v 0.31; P = .002) compared with BAL. However, complications were more common with lung biopsy (0.15 v 0.08; P = .006), and procedure-related mortality was four-fold higher for lung biopsy (0.0078) compared with BAL (0.0018). CONCLUSION: BAL may be the preferred diagnostic modality for the evaluation of potentially infectious pulmonary lesions because of lower complication and mortality rates; thus, choice of procedure depends on clinical suspicion of infection. Guidelines to promote consistency in the approach to the evaluation of lung infiltrates may improve clinical care of patients.


Assuntos
Aspergillus/isolamento & purificação , Biópsia , Lavagem Broncoalveolar , Transplante de Células-Tronco Hematopoéticas , Pneumopatias/diagnóstico , Pulmão/patologia , Neoplasias/cirurgia , Adulto , Aspergillus/genética , Biomarcadores/análise , Biópsia/efeitos adversos , Biópsia/mortalidade , Lavagem Broncoalveolar/efeitos adversos , Humanos , Pneumopatias/microbiologia , Pneumopatias/patologia , Neoplasias Pulmonares/diagnóstico , Mananas/análise , Pneumonia/diagnóstico , Pneumonia/microbiologia , Reação em Cadeia da Polimerase , Aspergilose Pulmonar/diagnóstico
9.
Br J Haematol ; 162(3): 376-82, 2013 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-23692048

RESUMO

Haemophagocytic lymphohistiocytosis (HLH) is a life threatening complication of Epstein-Barr virus (EBV) infection. The anti-CD20 antibody rituximab depletes B cells, leading to improved outcomes for patients with EBV-associated B-lymphoproliferative disorders. To gather data on the use of rituximab in EBV-HLH, we performed a retrospective investigation involving 42 EBV-HLH patients who had received treatment with rituximab-containing regimens. On average, patients received 3 rituximab infusions (range 1-10) at a median dose of 375 mg/m(2) . In all patients, rituximab was administered with other HLH-directed medications, including steroids, etoposide and/or ciclosporin. Rituximab-containing regimens appeared well tolerated and improved clinical status in 43% of patients. Examination of laboratory data obtained prior to and within 2-4 weeks after the first rituximab dose revealed significant reductions in EBV load (median load pre-rituximab: 114,200 copies/ml, median post-rituximab: 225 copies/ml, P = 0.0001) and serum ferritin levels (median ferritin pre-rituximab: 4260 µg/l, median post-rituximab: 1149 µg/l, P = 0.001). Thus, when combined with conventional HLH-directed therapies, rituximab improves symptoms, reduces viral load and diminishes inflammation. These data support the incorporation of rituximab into future prospective clinical trials for patients with EBV-HLH.


Assuntos
Anticorpos Monoclonais Murinos/uso terapêutico , Infecções por Vírus Epstein-Barr/complicações , Fatores Imunológicos/uso terapêutico , Linfo-Histiocitose Hemofagocítica/tratamento farmacológico , Linfo-Histiocitose Hemofagocítica/virologia , Adolescente , Adulto , Idoso , Anticorpos Monoclonais Murinos/administração & dosagem , Anticorpos Monoclonais Murinos/efeitos adversos , Antivirais/uso terapêutico , Criança , Pré-Escolar , Esquema de Medicação , Avaliação de Medicamentos/métodos , Quimioterapia Combinada , Infecções por Vírus Epstein-Barr/tratamento farmacológico , Infecções por Vírus Epstein-Barr/virologia , Feminino , Herpesvirus Humano 4/isolamento & purificação , Humanos , Fatores Imunológicos/administração & dosagem , Fatores Imunológicos/efeitos adversos , Masculino , Pessoa de Meia-Idade , Prognóstico , Estudos Retrospectivos , Rituximab , Análise de Sobrevida , Resultado do Tratamento , Carga Viral/efeitos dos fármacos , Adulto Jovem
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