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1.
Oncologist ; 25(2): e328-e334, 2020 02.
Artigo em Inglês | MEDLINE | ID: mdl-32043777

RESUMO

On May 24, 2019, the Food and Drug Administration approved ruxolitinib for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in adult and pediatric patients 12 years and older. Approval was based on Study INCB 18424-271 (REACH-1; NCT02953678), an open-label, single-arm, multicenter trial that included 49 patients with grades 2-4 SR-aGVHD occurring after allogeneic hematopoietic stem cell transplantation. Ruxolitinib was administered at 5 mg twice daily, with dose increases to 10 mg twice daily permitted after 3 days in the absence of toxicity. The Day-28 overall response rate was 57.1% (95% confidence interval [CI]: 42.2-71.2). The median duration of response was 0.5 months (95% CI: 0.3-2.7), and the median time from Day-28 response to either death or need for new therapy for acute GVHD was 5.7 months (95% CI: 2.2 to not estimable). Common adverse reactions included anemia, thrombocytopenia, neutropenia, infections, edema, bleeding, and elevated transaminases. Ruxolitinib is the first drug approved for treatment of SR-aGVHD. IMPLICATIONS FOR PRACTICE: Ruxolitinib is the first Food and Drug Administration-approved treatment for steroid-refractory acute graft-versus-host disease in adult and pediatric patients 12 years and older. Its approval provides a treatment option for the 60% of those patients who do not respond to steroid therapy.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Adulto , Criança , Doença Enxerto-Hospedeiro/tratamento farmacológico , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Nitrilas , Pirazóis/efeitos adversos , Pirimidinas , Esteroides/uso terapêutico
2.
Biol Blood Marrow Transplant ; 24(4): 758-764, 2018 04.
Artigo em Inglês | MEDLINE | ID: mdl-29196074

RESUMO

Acute kidney injury (AKI) is a common adverse event after hematopoietic cell transplantation (HCT). AKI is associated with early death or chronic kidney disease among transplant survivors. However, large-scale pediatric studies based on standardized criteria are lacking. We performed a retrospective analysis of 1057 pediatric patients who received allogeneic HCT to evaluate the incidence and risk factors of AKI according to AKI Network criteria within the first 100 days of HCT. We also determined the effect of AKI on patient survival. The 100-day cumulative incidences of all stages of AKI, stage 3 AKI, and AKI requiring renal replacement therapy (RRT) were 68.2% ± 1.4%, 25.0% ± 1.3%, and 7.6% ± .8%, respectively. Overall survival at 1 year was not different between patients without AKI and those with stage 1 or 2 AKI (66.1% versus 73.4% versus 63.9%, respectively) but was significantly different between patients without AKI and patients with stage 3 AKI with or without RRT requirement (66.1% versus 47.3% versus 7.5%, respectively; P < .001). Age, year of transplantation, donor type, sinusoidal obstruction syndrome (SOS), and acute graft-versus-host disease (GVHD) were independent risk factors for stages 1 through 3 AKI. Age, donor, conditioning regimen, number of HCTs, SOS, and acute GVHD were independent risk factors for AKI requiring RRT. Our study revealed that AKI was a prevalent adverse event, and severe stage 3 AKI, which was associated with reduced survival, was common after pediatric allogeneic HCT. All patients receiving allogeneic HCT, especially those with multiple risk factors, require careful renal monitoring according to standardized criteria to minimize nephrotoxic insults.


Assuntos
Injúria Renal Aguda/microbiologia , Transplante de Células-Tronco Hematopoéticas , Doença Aguda , Injúria Renal Aguda/etiologia , Injúria Renal Aguda/terapia , Adolescente , Adulto , Fatores Etários , Criança , Pré-Escolar , Intervalo Livre de Doença , Feminino , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/mortalidade , Doença Enxerto-Hospedeiro/terapia , Humanos , Lactente , Masculino , Terapia de Substituição Renal , Estudos Retrospectivos , Fatores de Risco , Taxa de Sobrevida
3.
Pediatr Blood Cancer ; 63(12): 2078-2085, 2016 12.
Artigo em Inglês | MEDLINE | ID: mdl-27535002

RESUMO

Patients with acute myeloid leukemia (AML) have relatively low survival rates compared to patients with other pediatric cancers. Relapse is frequent with conventional treatment and is a major cause of morbidity and mortality. Natural killer (NK) cells offer an alternative approach to chemotherapy that combats relapse by substantially eradicating AML blasts. New methods for enhancing NK cell activation and expression of the activating ligand on target malignant cells will increase the likelihood of success with this approach. We review these latest discoveries in NK cell-based therapy for AML and delineate recent advances in sensitizing AML cells to NK cell-mediated immunosurveillance.


Assuntos
Imunoterapia/métodos , Células Matadoras Naturais/imunologia , Leucemia Mieloide Aguda/terapia , Humanos , Leucemia Mieloide Aguda/imunologia , Ativação Linfocitária
4.
J Pediatr Hematol Oncol ; 30(8): 612-7, 2008 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-18799940

RESUMO

Hypocalcemia is a rare complication of osteosarcoma, having been described in only 4 reports. We present the case of a 16-year-old male with metastatic osteosarcoma of the right humerus who was found to have severe asymptomatic hypocalcemia. Cytogenetic analysis of peripheral blood revealed a microdeletion in band 22q11.2. Following amputation of the tumor-bearing extremity, the patient's calcium levels increased, but did not normalize. These findings suggested that the etiology of his hypocalcemia was osteoblastic utilization of calcium by the tumor, exacerbated by 22q11.2 deletion syndrome.


Assuntos
Neoplasias Ósseas/complicações , Síndrome de DiGeorge/complicações , Hipocalcemia/etiologia , Osteossarcoma/complicações , Absorciometria de Fóton , Adolescente , Amputação Cirúrgica , Neoplasias Ósseas/patologia , Neoplasias Ósseas/fisiopatologia , Síndrome de DiGeorge/patologia , Síndrome de DiGeorge/fisiopatologia , Humanos , Úmero/patologia , Úmero/cirurgia , Hipocalcemia/patologia , Hipocalcemia/fisiopatologia , Hibridização In Situ , Masculino , Osteossarcoma/patologia , Osteossarcoma/fisiopatologia
5.
Semin Hematol ; 52(3): 215-22, 2015 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-26111469

RESUMO

Core binding factor (CBF) is a heterodimeric protein complex involved in the transcriptional regulation of normal hematopoiesis. Mutations in CBF-encoding genes result in leukemogenic proliferative advantages and impaired differentiation of the hematopoietic progenitors. CBF molecular aberrations are responsible for approximately 20% of all adult acute myeloid leukemia (AML). Although CBF-AMLs are considered to have relatively good prognosis compared to other leukemia subtypes, they are a heterogeneous group of disorders and modern therapy frequently leads to relapse and the associated morbidity and mortality. Improvements in risk stratification and development of targeted therapies are needed for better outcomes. In this review we provide a brief overview of the molecular basis, prognostic categories and the advanced treatment strategies for CBF leukemias.


Assuntos
Fatores de Ligação ao Core/imunologia , Leucemia Mieloide Aguda/diagnóstico , Animais , Humanos , Leucemia Mieloide Aguda/genética , Leucemia Mieloide Aguda/imunologia , Mutação , Prognóstico , Resultado do Tratamento
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