The American journal of pediatric hematology/oncology最新文献

Purpose: We evaluated the antiemetic efficacy of tropisetron, a 5-HT3 receptor antagonist, during its compassionate use in children with malignant disease who during previous chemotherapy cycles experienced emesis refractory to metoclopramide-based treatments.

Patients and methods: Tropisetron was given to 15 children (eight boys and seven girls 18 months to 18 years of age) with miscellaneous neoplasms. Generally 5 mg/day of tropisetron was administered i.v. the first day of cisplatin-based chemotherapy and i.v. or orally each subsequent day of chemotherapy. The dose of tropisetron was reduced to 2 mg/day in children < 2 years of age and weighing < 20 kg.

Results: Vomiting was well controlled (no more than two episodes per day) on 118 of the 184 days of treatment with tropisetron (64%). No clinically important variations were observed in blood pressure, heart rate, body temperature, or electrocardiographic findings attributable to tropisetron. Transient, mild to moderate side effects (headache, constipation, abdominal pain, diarrhea) occurred in five patients on 11 of the 184 days of tropisetron treatment (6%).

Conclusion: The results obtained during compassionate use of tropisetron confirm that it is a valid, safe, and manageable antiemetic for the treatment of pediatric patients.

Patients and methods: Seven children of the same family with a possible variant of Diamond-Blackfan anemia (DBA) are reported. Five were male siblings, and the other two were their cousins, one male and one female. All were products of consanguineous marriages of healthy parents. All cases occurred within one generation. Anemia was present at birth or shortly after birth. Hepatosplenomegaly was present in all. Four had short stature. Hematological findings included normochromic, normocytic, or macrocytic anemia, marked reticulocytopenia, with initial normal white blood cell and platelet count, and absent or markedly decreased erythroid precursors on bone marrow examination. All were treated initially with prednisolone; in one patient oxymetholone was added.

Results: Three children failed to respond to the initial treatment, and also failed to respond to cyclosporin A and pulse doses of methylprednisolone. Myelofibrosis occurred in two siblings, 9 and 11 years from diagnosis. In two children the disease recurred 9 and 12 years after initial diagnosis.

Conclusions: Our cases point to a possible variant of DBA characterized by the presence of normochromic normocytic anemia, hepatosplenomegaly, absent skeletal malformations, and unusual long- term complications.

Purpose: Little information is available regarding splenic injury in patients with hemophilia. We describe here the management of splenic rupture in five of our patients with hemophilia and summarize the literature describing this complication.

Patients and methods: Two human immunodeficiency virus-seropositive patients were managed medically and did not require splenectomy. A third patient had a high titer inhibitor to both porcine and human factor VIII and required emergency splenectomy. Two boys had not been previously diagnosed with hemophilia until they underwent splenectomy after abdominal trauma.

Results: All five patients survived.

Conclusions: These cases demonstrate that nonsurgical management of splenic injury in patients with hemophilia can be performed safely and that splenectomy can be successfully performed despite a high titer of factor VIII inhibitor.

Purpose: To evaluate the kinetics of erythropoietin (EPO) production and address the pathogenesis of anemia of prematurity, we measured EPO levels in infants during the first year of life.

Patients and methods: Using a radioimmunoassay, serum EPO levels were measured in 97 infants classified into three groups according to weight: group A, n = 40, < 1,500 g; group B, n = 19, 1,500-2,499 g; and group C, n = 38, > or = 2,500 g.

Results: The serum EPO level ranged widely during the early neonatal period from days 0 to 6 (group A, < 5 to 307 mU/ml; group B, 10-340 mU/ml; and group C, 9-108 mU/ml). EPO reached its lowest level (< 20 mU/ml) between days 7 and 50 in all groups. The hemoglobin concentration reached its nadir between days 51 and 150 in all groups, with the lowest concentration observed in low birth weight infants. In contrast, the EPO level during the anemic phase was approximately 20 mU/ml and was independent of birth weight. A negative correlation between serum EPO level and hemoglobin concentration was observed only in group C (r = -0.54, p < 0.05). The negative slope of the regression equation in group C exceeded that of groups A and B (p < 0.05). When the relationship between EPO and Hb was evaluated over periods of 7-50 days, 51-100 days, and > 101 days, respectively, we noted a significant correlation between values on days 7 and 50 in group A (r = -0.53, p < 0.05) and between days 51 and 100 in group B (r = -0.76, p < 0.05).

Conclusions: These data suggest the appreciable EPO production in premature infants, but its insufficient response to the depressed hemoglobin level, implying the need to administer exogenous EPO to infants with anemia of prematurity.

Purpose: The purpose of this case report is to (a) present a detailed description of a child with sickle cell anemia who developed sarcoidosis, (b) compare the clinical aspects of sickle cell anemia and sarcoidosis, and (c) review the literature with respect to the coincidence of these two diseases. Detailed clinical case history and laboratory findings are presented. A literature review of other case reports with children and adults who have both sickle cell anemia and sarcoidosis is given.

Results: Based on limited data, there may be an increased incidence of sarcoidosis in adults with sickle cell diseases. No data are available in the pediatric population.

Conclusions: Sarcoidosis is difficult to diagnose in children with sickle cell anemia. The clinical manifestations of sickle cell anemia and sarcoidosis have overlapping features but represent distinct syndromes. Patients with sickle cell anemia who have unusual signs and symptoms need to be further evaluated for the possibility of other coincident diseases, including sarcoidosis.

Purpose: Reports from the National Wilms' Tumor Study (NWTS) Group on the subject of chest computed tomography (CT) versus chest radiograph for the detection of lung metastases from Wilms tumor are reviewed.

Patients and methods: Thirty-two patients with lung nodules detected by CT, with negative chest radiographs, were identified. Five patients were excluded from further analysis. Of the remaining 27 patients, 18 were treated as stage IV, receiving therapy with three drugs and lung irradiation; the other nine were treated with less intensive therapy and no lung irradiation. The investigators found no significant difference between the overall survival between these two groups of patients (94% and 88%, respectively). In an earlier study, four of 11 children (36%) with normal chest radiographs and positive chest CT results were treated by ignoring the CT findings. These data compared with a relapse rate of only 20% in a control population.

Results: The studies do not statistically address the question of the impact of CT of the chest on survival.

Conclusions: All children should have the benefit of the most sensitive imaging available, including CT, to detect tumor spread.

Purpose: To determine the relationship between hematological toxicity and actual dose intensity of treatment of patients randomized to therapy during the first 28 months of the National Wilms' Tumor Study-4.

Methods: The mean minimum white blood cell count (WBC), platelet count (PLT), hemoglobin, and absolute neutrophil count (ANC) during the first two courses of chemotherapy and the mean number of days of hospitalization for toxicity were compared between standard and "pulse-intensive" regimens for all randomized patients entered on National Wilms' Tumor Study-4 between August 6, 1986 and December 31, 1988. The mean dose intensity of dactinomycin, vincristine, and doxorubicin received during the first two courses and the entire course of treatment was compared between standard and "pulse-intensive" regimens.

Results: The mean minimum WBC, PLT, and ANC were all significantly lower during the first two courses of chemotherapy for stage I patients treated with the standard regimen, compared with the "pulse-intensive" regimen. The mean dose intensity of dactinomycin and doxorubicin was significantly higher for patients treated with the "pulse-intensive" regimens, compared with the appropriate standard regimen.

Conclusions: The "pulse-intensive" administration schedule for the treatment of children with Wilms' tumor permits administration of chemotherapy at a higher dose intensity without an increase in hematological toxicity.

Purpose: Iron deficiency anemia (IDA) causes benign intracranial hypertension (BIH).

Patients and methods: A case of an 11-year-old girl with severe IDA and benign intracranial hypertension is presented.

Results: Treatment of iron deficiency resulted in the resolution of BIH. Recurrence of BIH is observed with the recurrence of IDA. Chronic intermittent blood loss from the intestinal polyps was the etiology of recurrent IDA.

Conclusions: A thorough search to determine the etiology of IDA is essential for proper treatment. Although BIH is a rare complication of IDA, its association with IDA should be recognized for prompt treatment before an extensive and costly workup for BIH is done.

Purpose: We describe for the first time the case of a child with Kawasaki syndrome and associated transient erythroblastopenia.

Patients and methods: A 5 1/2-month-old infant with Kawasaki syndrome as evidenced by lymphadenopathy, fever, rash conjunctival injection, and extremity changes had associated anemia and reticulocytopenia requiring transfusion and thrombocytopenia. Bone marrow aspiration was consistent with a transient erythroblastopenia. She developed cardiac aneurysms despite therapy with i.v. immunoglobulin and aspirin.

Results: The anemia and reticulocytopenia resolved with normalization of her hemoglobin. The platelet count increased and she developed a thrombocytosis characteristic of this clinical entity. She completely recovered without recurrence of either the anemia or reticulocytopenia.

Conclusions: We speculate that the cause of the erythroblastopenia and thrombocytopenia is an inflammatory insult of Kawasaki syndrome on the bone marrow and its various lineages causing the myriad hematological abnormalities now associated with Kawasaki syndrome. It is possible that the i.v. immunoglobulin aids in neutralizing the triggering agent and therefore shortening the duration of the marrow insult.

Purpose: During a 6-year period, we identified six infants < 6 months of age with transient erythroblastopenia of childhood (TEC).

Patients and methods: All patients presented with moderate to severe anemia, reticulocytopenia, and age-appropriate mean corpuscular volume and fetal hemoglobin level.

Results: Severe aregenerative anemia in an infant < 6 months of age often raises the diagnostic possibility of Diamond Blackfan anemia (DBA). Given prognostic and therapeutic implications, distinction between DBA and TEC is of crucial importance.

Conclusions: We suggest that TEC may occur more commonly in infants < 6 months of age than has heretofore been recognized and that it has a clinical and hematologic picture similar to that seen in older children. Recognition of the occurrence of TEC in very young infants may help avoid an inappropriate diagnosis of DBA.