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1.
J Pediatr Hematol Oncol ; 44(3): e728-e732, 2022 Apr 01.
Artigo em Inglês | MEDLINE | ID: mdl-34133379

RESUMO

Vanishing bile duct syndrome is a rare paraneoplastic syndrome occasionally seen in pediatric Hodgkin lymphoma. It is usually regarded as a fatal disorder. Here, we present a case of vanishing bile duct syndrome cholestasis related to Hodgkin lymphoma that resolved after chemotherapy and radiation.


Assuntos
Colestase , Doença de Hodgkin , Síndromes Paraneoplásicas , Protocolos de Quimioterapia Combinada Antineoplásica/efeitos adversos , Ductos Biliares/patologia , Criança , Colestase/etiologia , Doença de Hodgkin/complicações , Doença de Hodgkin/tratamento farmacológico , Doença de Hodgkin/patologia , Humanos , Síndromes Paraneoplásicas/tratamento farmacológico , Síndromes Paraneoplásicas/etiologia
2.
Pediatr Hematol Oncol ; 37(6): 465-474, 2020 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-32338562

RESUMO

Background: Panobinostat demonstrates activity against pediatric cancers in vitro. A phase I trial in children with refractory hematologic malignancies was conducted. Study design: The trial evaluated two schedules of oral panobinostat using 3 + 3 dose escalations in 28-day cycles. For children with leukemia, panobinostat was given once daily three days a week each week at 24, 30 and 34 mg/m2/day. For children with lymphoma, panobinostat was given once daily three days a week every other week at 16, 20 and 24 mg/m2/day. Cerebrospinal fluid (CSF) from Day 29 of the first cycle, when available, was evaluated for PK. The study was registered on clinicaltrials.gov (NCT01321346) Results: Twenty-two subjects enrolled with leukemia. Five enrolled at dose level 1, 6 at dose level 2, and 11 at dose level 3. There was one dose limiting toxicity (DLT) in the leukemia arm at dose level 3 (Grade 4 hypertriglyceridemia), but no maximum tolerated dose (MTD) was identified. No subjects required removal from protocol therapy for QTc prolongation. PK studies were available in 11 subjects with similar exposure in children as in adults. Four Day 29 CSF specimens were found to have panobinostat levels below the lower limit of quantification. Five subjects with lymphoma were enrolled and received study drug, and 4 were evaluable for DLT. A DLT was reported (Grade 3 enteritis) on the lymphoma arm. Conclusions: Panobinostat was tolerated in heavily pretreated pediatric subjects. Gastrointestinal effects were observed on this study. There were no cardiac findings. There were no responses.


Assuntos
Neoplasias Hematológicas/tratamento farmacológico , Leucemia/tratamento farmacológico , Linfoma/tratamento farmacológico , Panobinostat/administração & dosagem , Administração Oral , Adulto , Criança , Feminino , Neoplasias Hematológicas/sangue , Humanos , Hipercolesterolemia/sangue , Hipercolesterolemia/induzido quimicamente , Leucemia/sangue , Linfoma/sangue , Masculino , Panobinostat/efeitos adversos , Recidiva
5.
J Pediatr Hematol Oncol ; 30(2): 166-71, 2008 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-18376272

RESUMO

Severe congenital protein C deficiency is a rare life-threatening disorder that presents with purpura fulminans, disseminated intravascular coagulation, and thrombotic complications during the neonatal period. Affected children require acute replacement therapy with fresh frozen plasma or protein C concentrate, for example, Ceprotin (Baxter AG, Vienna). Long-term management and outcome is dependent on effective anticoagulation with warfarin, low-molecular weight heparin, or protein C concentrate. We describe the successful use of intravenous protein C concentrate for thrombotic prophylaxis in 2 sisters with severe type I protein C deficiency. Individualized long-term prophylactic regimens were developed based on clinical response. In vivo pharmacokinetic analyses of protein C concentrate were performed in each patient. Analysis of the protein C gene coding sequences identified 2 mutations in both patients, the previously described Arg169 to Trp mutation, and a novel mutation that changes Cys17 into a stop codon.


Assuntos
Deficiência de Proteína C/genética , Proteína C/genética , Proteína C/uso terapêutico , Sequência de Bases , Feminino , Humanos , Recém-Nascido , Dados de Sequência Molecular , Mutação , Proteína C/farmacocinética
6.
Pediatr Blood Cancer ; 47(2): 218-23, 2006 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-16086426

RESUMO

Post-transplant lymphoproliferative disease (PTLD) is a well-recognized complication of the intense immunosuppression required in solid organ and bone marrow transplant recipients. The clinical presentation is varied and can range from a benign infectious mononucleosis-like syndrome to malignant lymphoma. PTLD manifesting as multiple myeloma occurs rarely. We report the unique occurrence of Epstein-Barr virus (EBV)-associated post-transplant multiple myeloma in a 16-year-old male. In contrast to previously described cases of PTLD-myeloma type, this patient was very young, had a clear association with EBV, and an indolent clinical course.


Assuntos
Herpesvirus Humano 4 , Transplante de Rim/efeitos adversos , Mieloma Múltiplo/etiologia , Adolescente , Transplante de Coração/efeitos adversos , Humanos , Terapia de Imunossupressão/efeitos adversos , Masculino , Mieloma Múltiplo/patologia , Mieloma Múltiplo/virologia
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