Journal of pediatric hematology/oncology
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J. Pediatr. Hematol. Oncol. · Jan 2011
Case ReportsDevelopment of T-cell acute lymphoblastic leukemia in a patient in very long lasting complete remission of juvenile myelomonocytic leukemia.
Juvenile myelomonocytic leukemia (JMML) occurs with an incidence of 1.2 per million children a year, and represents 18% to 30% of all myelodysplastic (MDS) and myeloproliferative (MPS) disorders in the age group below 15, being by far the most common MDS/MPS in children younger than 4 years. The only therapeutic approach which results in a definitive cure of patients with JMML is myeloablative chemo-therapy/radio-therapy, followed by allogeneic hematopoietic cell transplantation. Few cases of transformation of JMML in acute lymphoblastic leukemia have been reported. ⋯ Treatment with acute lymphoblastic leukemia (ALL)-directed chemotherapy induced complete restoration of normal hemopoiesis, but testicular involvement persisted. The patient died after transplantation with unrelated cord blood. This case suggests that JMML is a true stem cell disorder and that stem cell transplantation should be considered, even in patients with a very favorable clinical course.
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J. Pediatr. Hematol. Oncol. · Nov 2010
Review Case ReportsTreatment of Gorham-Stout disease with zoledronic acid and interferon-α: a case report and literature review.
Gorham-Stout disease is a rare disease characterized by osteolysis, angiomatosis, and soft-tissue swelling. It is a diagnosis of exclusion and has an unknown etiology. ⋯ There is no standard of treatment. We report a case of a 16-year-old female with Gorham-Stout disease and recurrent pleural effusions who was successfully treated with concurrent zoledronic acid and peg-interferon α-2b.
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J. Pediatr. Hematol. Oncol. · Nov 2010
Early changes in SOFA score as a prognostic factor in pediatric oncology patients requiring mechanical ventilatory support.
To evaluate whether changes in outcome prediction scores during the first 72 hours after admission to a pediatric intensive care unit (PICU) are more predictive of outcome than single assessments at admission in pediatric oncology patients requiring mechanical ventilatory support for more than 3 days. ⋯ Serial evaluation of SOFA score during the first few days after PICU admission was a good predictor of prognosis in pediatric oncology patients mechanically ventilated over 3 days. Independent of initial SOFA score, Δ-SOFA score during the first 72 hours closely correlated with outcome. Early changes in respiratory parameters, such as P/F ratio, OI, and VI, may also provide valuable prognostic information in such patients.
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J. Pediatr. Hematol. Oncol. · Oct 2010
Randomized Controlled TrialA family-based randomized controlled trial of pain intervention for adolescents with sickle cell disease.
The study had 2 aims---to determine the efficacy of a family-based cognitive-behavioral pain management intervention for adolescents with sickle cell disease (SCD) in (1) reducing pain and improving health-related variables and (2) improving psychosocial outcomes. Each adolescent and a family support person were randomly assigned to receive a brief pain intervention (PAIN) (n=27) or a disease education attention control intervention (DISEASE ED) (n=26) delivered at home. Assessment of primary pain and health-related variables (health service use, pain coping, pain-related hindrance of goals) and secondary psychosocial outcomes (disease knowledge, disease self-efficacy, and family communication) occurred at baseline (before randomization), postintervention, and 1-year follow-up. ⋯ Efforts to address barriers to participation and improve feasibility of psychosocial interventions for pediatric SCD are critical to advancing development of effective treatments for pain. Sample size was insufficient to adequately test efficacy, and analyses did not support this focused cognitive-behavioral pain management intervention in this sample of adolescents with SCD. Exploratory analyses suggest that comprehensive interventions, that address a broad range of skills related to disease management and adolescent health concerns, may be more effective in supporting teens during healthcare transition.
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J. Pediatr. Hematol. Oncol. · Oct 2010
Macrophage activation syndrome in the acute phase of Kawasaki disease.
Rare cases of macrophage activation syndrome (MAS) occurring during the acute phase of Kawasaki disease (KD) have been reported. We sought to characterize, review treatment, and outcomes of KD patients with clinical features of MAS. Medical histories of patients treated for KD and MAS between January 2001 and March 2008 were reviewed. ⋯ Treatment beyond the standard KD protocol (aspirin + intravenous immunoglobulin) was necessary in all but 1 patient. All patients eventually recovered with no long-term sequelae. A high index of suspicion for clinical features associated with MAS is warranted for KD patients to provide appropriate and timely treatment.