The American journal of pediatric hematology/oncology最新文献

Purpose: In order to assist in the management of newly diagnosed children with moderate aplastic anemia (MAA) we reviewed the clinical course and outcome of children with MAA seen at our institution over the past 12 years and compared them with children with severe aplastic anemia (SAA).

Patients and methods: MAA was defined as having a hypocellular bone marrow and cytopenia in at least two cell lines not in the severe range. Twelve children met these criteria. Twenty-eight children with SAA were seen during the same interval. Patients with MAA were treated with immunomodulation with antithymocyte globulin and/or cyclosporine if they progressed to SAA.

Results: Five patients with MAA progressed to SAA at a median interval of 18 months from diagnosis. The other seven patients required no therapy or only received transfusions for < or = 6 months after diagnosis. The survival of the patients with MAA was significantly better than that of patients with SAA treated with immunomodulation (p = 0.022). All patients with MAA are alive at a median follow up of 7 years and are transfusion independent; only one patient currently receives therapy. Residual hematologic abnormalities in children with MAA included thrombocytopenia, leukopenia, and macrocytosis.

Conclusions: In this small series of children with MAA the outcome was excellent and significantly better than in patients with SAA; more than half recovered with minimal or no therapy. Patients who progressed to SAA responded well to treatment. A larger prospective study is needed to conclusively define the natural history of MAA.

Purpose: Six children who had mature or immature sacrococcygeal teratomas diagnosed in the newborn period have since been registered on a Pediatric Oncology Group/Children's Cancer Group germ cell study with recurrent malignant neoplasia [pure yolk sac tumor (YST) or teratoma with yolk sac elements].

Results: Four of the children have responded to therapy, one has died, and one has been lost to follow-up. Review of the slides from five of the original tumors identified microscopic foci of YST in four.

Conclusions: Detection of such foci in neonatal tumors is important because serum alpha-fetoprotein concentrations may not be helpful since they may normally be high in the newborn period due to fetal production.

Purpose: We describe a poorly differentiated adenocarcinoma of the lung in an 11-year-old boy who had had a pulmonary hamartoma for at least 8 years.

Conclusions: A hamartoma, even if clinically silent, may lead to the development of malignant tumors on rare occasions.

Purpose: T cell-mediated red cell aplasia in a 4 1/2-year-old child with transient erythroblastopenia of childhood (TEC) is described.

Patients and methods: Erythropoiesis was studied by assessing the colony growth of marrow erythroid progenitors at the time of diagnosis and during recovery.

Results: The colony-forming unit-erythroid (CFU-E) growth of whole marrow at diagnosis was only 28% that of the control. T-cell depletion of the patient's marrow was followed by a more than fivefold increase in CFU-E growth, as compared with 20% inhibition of CFU-E and 40% inhibition of burst-forming unit-erythroid (BFU-E) growth in control marrow. The number of colony-forming unit-granulocyte-macrophage (CFU-GM) in both control and patient's marrow was not significantly altered by all of these manipulations. During early and late recovery, CFU-E and BFU-E growth improved substantially, and the effect of T-cell depletion diminished. Increased numbers of peripheral T-suppressor lymphocytes, as well as activation of natural killer (NK) cells and high levels of interferon, all consistent with viral infection, were found at presentation. Clinical recovery was associated with normalization of T-suppressor lymphocyte number.

Conclusions: The results suggest that in this child with TEC, a preceding viral infection may have caused activation of suppressor T-cells and interferon secretion leading to cell-mediated suppression of erythropoiesis.

Purpose: Five patients with Fanconi anemia have been treated by bone marrow transplantation.

Patients and methods: They were conditioned with cyclophosphamide (CY) (20-150 mg/kg), antilymphocyte globulin, and thoracoabdominal irradiation (4-6 Gy). The dose of CY for preconditioning was adjusted individually, based on the in vitro effect of CY metabolites on the chromosomes of patients with Fanconi anemia. Four patients received marrow from human leukocyte antigen (HLA)-identical siblings, and one received marrow from his HLA phenotypically identical father.

Results: All patients achieved engraftment, and acute graft-versus-host disease (GVHD) grade II or more was not observed. Three developed chronic GVHD. All patients are surviving 2-5 years after grafting, with hematological improvement.

Conclusions: These results indicate that the individual dose adjustment of CY used for preconditioning may prevent graft failure and severe acute GVHD.

Purpose: We report here our experience in using intravenous methohexital (MHX), an ultrashort-acting barbiturate, for brief unconscious sedation of pediatric oncology outpatients undergoing painful, invasive procedures.

Methods: Following published monitoring guidelines for deep pediatric sedation, 1.0 mg/kg MHX was administered immediately before the procedure, 1% xylocaine was given locally, and MHX was additionally titrated to maintain minimal response to pain during the procedure. Clinical data reported here were gathered retrospectively from permanent medical records.

Results: Data reported here represent 132 evaluable consecutive procedures in 33 patients ranging in age from 1.6 to 20.5 years. Patients underwent an average of 4 +/- 3 procedures and received a mean total MHX dose per procedure of 5.8 +/- 2.1 mg/kg. The mean length of time from start of sedation to full arousability was 30 +/- 12 min. Twenty-three (17.4%) procedures were associated with clinically insignificant decreases in diastolic blood pressure or heart rate below resting normal ranges for age. Eight (6.1%) procedures in six patients were associated with minor complications requiring no intervention, such as transient behavioral changes, transient myoclonus, and minimal stridor. Five procedures (3.8%) in five patients required simple suctioning to manage secretions. Only two procedures (1.5%) in two patients required brief bag-mask ventilation plus suctioning for suspected laryngospasm. None required intubation. No differences in clinical features or MHX doses were noted for patients with, as compared to those without, complications. All procedures were completed with a satisfactory level of sedation.

Conclusions: Our experience indicates that MHX, with appropriate monitoring as described here, is a safe and effective agent for use in pediatric oncology outpatient sedation programs.

Purpose: A case of alveolar soft part sarcoma of the arm with metastatic pulmonary miliaria is reported in a 12-year-old girl.

Results: Although the size of the metastases increased greatly and progressively for 9 years with no improvement under chemotherapy, the patient's general condition remained good.

Conclusions: The course in pediatric oncology of this rare mesenchymatous tumor is very unusual; the histologic pattern is characteristic but the histogenesis remains unclear. The treatment of choice is tumoral excision. Other therapies involving secondary deposits are far from satisfactory, and the prognosis is poor because of the high rate of metastases.

Purpose: 13-cis-Retinoic acid (cis-RA) has efficacy in the treatment and prevention of certain malignancies. In vitro effects against neuroblastoma include induction of differentiation, inhibition of proliferation, and decreased N-myc expression. We hypothesized that cis-RA may be effective against minimal residual disease in neuroblastoma patients. A phase I trial to determine the maximal tolerated dosage and toxicity of cis-RA in pediatric patients with neuroblastoma after bone marrow transplantation was initiated.

Patients and methods: Forty-nine pediatric patients (status post-bone marrow transplant for neuroblastoma) were treated for 14 days with oral cis-RA in escalating doses from 100 to 200 mg/m2/day followed by a 14-day rest period for up to 12 months.

Results: In three of 39 patients (7.7%) evaluable for calcium levels, hypercalcemia (12.6-18.7 mg/dl) was the dose-limiting toxicity. Grade 1-3 hypercalcemia occurred in nine of 39 patients (23%). The overall incidence of hypercalcemia was 31% (12 of 39). Only one patient was symptomatic due to the hypercalcemia, with arthralgias and myalgias. The hypercalcemia resolved with temporary discontinuation of the drug and a 25% dose reduction for subsequent courses.

Conclusions: Hypercalcemia is a novel dose-limiting toxicity for cis-RA. Patients receiving high doses of cis-RA should have monitoring of serum calcium levels.

Purpose: An 8-year-old boy with mild hemophilia A and inhibitors developed an acute myocardial infarction during treatment with prothrombin complex concentrates.

Conclusions: This rare complication warrants restriction of dosage and length of treatment with these products to the recommended guidelines. It also appears that noninvasive cardiac monitoring of these patients is beneficial.