The American journal of pediatric hematology/oncology最新文献

Purpose: Acute splenic sequestration in children with sickle hemoglobinopathies is a medical emergency characterized by splenomegaly, anemia, and thrombocytopenia.

Patients and methods: We describe a young girl with hemoglobin SC disease who experienced an acute splenic sequestration without palpable splenomegaly.

Conclusions: We emphasize the need for a high index of suspicion for splenic sequestration and the utility of ultrasonography in documenting splenomegaly.

Purpose: We report that the use of alternate-day cyclosporine and prednisone improved the clinical course of a 6-year-old child with severe Evans syndrome. Before the use of cyclosporine the child had experienced life-threatening episodes of hemolytic anemia despite the use of multiple therapeutic modalities.

Methods: Cyclosporine was given at a dose of 10 mg/kg/day divided into two doses on alternate days.

Results: The use of cyclosporine resulted in increased hemoglobin levels, increased platelet counts, and the reduction of the patient's prednisone dose from 2 mg/kg/day to as low as 1 mg/kg every other day. With this regimen, the patient had less severe hemolytic anemia, was less thrombocytopenic, and had fewer hospitalizations. No major toxic effects were associated with cyclosporine therapy.

Conclusion: The regimen of alternate-day cyclosporine and prednisone may prove to be useful in the treatment of other patients with refractory Evans syndrome.

Purpose: The goals of this study were (a) to determine the number of peripheral blood burst forming units-erythroid (BFU-E); (b) to define the relationship between circulating BFU-E number and fetal hemoglobin (HbF) level; and (c) to define the relationship between BFU-E number and age in pediatric sickle cell disease (SCD) patients.

Patients and methods: Fetal hemoglobin (HbF) level and peripheral blood BFU-E number were determined in children < 18 years of age with SCD in a steady state of their disease. These data were compared with those of normal children.

Results: An increased number of BFU-E was observed in the peripheral blood of children with SCD compared with normals (30.7 vs. 15.7 per 10(5) mononuclear cells, respectively; p = 0.009). Overall there was the suggestion of a direct relationship between HbF level and peripheral blood BFU-E number (regression coefficient = 0.445; p = 0.06). Additionally, a strong inverse relationship between BFU-E number and age (regression coefficient = -0.671; p < 0.0001) was observed.

Conclusions: In children with SCD (a) there are an increased number of peripheral blood BFU-E compared with normal children; (b) the inverse relationship between HbF level and BFU-E number observed in adult SCD patients is not seen in children; and (c) there is a strong inverse relationship between age and BFU-E number. This information may help to further clarify the relationship between peripheral blood BFU-E and erythropoietic stress.

Purpose: The preclinical detection of neuroblastoma by screening for elevated levels of urinary catecholamines often leads to the discovery of children with early-stage, biologically favorable disease. It is uncertain how vigorously therapy must be pursued in such cases. We report an infant whose pelvic mass was initially thought to be a fecaloma, and consequently was not treated for 19 months.

Case report: A 2-month-old girl was referred for evaluation for the presence of a neuroblastoma because of elevated urinary catecholamines detected in a mass screening program. Although no mass was initially found, urinary catecholamines became increasingly elevated, and a pelvic mass was finally radiologically identified. It was resected. Histology showed it to be a neuroblastoma, POG stage C, International stage 3, with unfavorable Shimada features, although near triploid with an unamplified N-myc oncogene. Chemotherapy was given for five cycles, and the child remains well, with no evidence of disease.

Conclusion: Although mass screening programs often detect early- stage, biologically favorable neuroblastomas that may spontaneously regress, our case had rising levels of urinary catecholamines and unfavorable histologic features at the time of resection. The unusual location of the tumor, and radiologic features of a fecaloma, contributed to the delay in definitive diagnosis, although persistence of elevated urinary catecholamines in the absence of a tumor is infrequent.

Purpose: Magnetic resonance imaging (MRI) was used for evaluation of refractory joint swelling in a 7-year-old boy with hemophilia.

Patient and methods: This patient with no evidence of an inhibitory had refractory left knee swelling despite receiving appropriate factor VIII concentrate infusions.

Results: A plain radiograph showed soft tissue swelling and a calcification in the left suprapatellar region. On MRI a discrete suprapatellar pouch could be detected and was subsequently surgically resected.

Conclusion: We believe that MRI should be considered when evaluating children with hemophilia who have refractory joint swelling and no evidence of an inhibitor.

Purpose: A multitude of risk factors has been described for patients with neuroblastoma. Little is known about the mutual interrelationship of these factors and their impact on patients with localized disease only.

Patients and methods: We investigated the possible influence of 37 variables univariately on event-free survival (EFS) in 308 consecutive patients with neuroblastoma stages I-III using Kaplan-Meier estimates. The chi 2 test was applied to detect nonrandom correlations, and the Cox's regression model was used for the multivate evaluation of identified factors.

Results: Seventeen factors appeared to influence EFS in stage I-III patients (p < 0.05, log-rank > 3.84), whereas 10 factors were found in the subgroup of stage III patients with midline crossing tumors (= stage III*, n = 128). The majority of univariately identified risk factors showed a nonrandom correlation to several others (p < 0.05). The multivariate analysis according to Cox selected for the patients with stages I-III the factors lactate dehydrogenase (LDH) (p = 0.0011), resectability (p = 0.0167), weight loss (p = 0.0185), tumor extension beyond midline (p = 0.0207), and age (p = 0.0233). For stage III* patients the model identified the factors LDH (p = 0.0089), weight loss (p = 0.0135), resectability (p = 0.0408), and age (p = 0.0700). The identification of these independent risk factors permitted the description of risk groups with EFS ratios after > 6 years between 22% and 96%.

Conclusions: Risk estimation of high discriminating power is possible for patients with localized neuroblastoma using simple, readily available clinical data.

Purpose: We report on the safety and efficacy of heparin anticoagulation for the treatment of neonatal renal vein thrombosis.

Patients and methods: Six consecutive, prospectively identified, critically ill neonates with renal vein thrombosis were studied. Diagnosis of renal vein thrombosis was based on history and examination and confirmed with renal ultrasound. All neonates were treated with continuous i.v. heparin titrated to achieve a therapeutic whole blood clotting time and/or APTT.

Results: Renal vein thrombosis was bilateral for three of six neonates. Heparin infusion rates varied from 8 to 40 U/kg/h and were administered for 7-14 days. Two neonates developed hemorrhagic complications; one had disseminated intravascular coagulation but did not hemorrhage until heparin toxicity ensued, and another was well until an umbilical catheter was removed while he was therapeutically heparinized. Renal outcome at 3 months to 6 years showed hypertension in one neonate, atrophic kidneys in two, and both hypertension and an atrophic kidney in one.

Conclusions: Bleeding was a significant complication of heparin therapy for neonatal renal vein thrombosis. Renal dysfunction was not prevented in four of six neonates treated with heparin. Alternative approaches to titrate heparin, alternative anticoagulants, or fibrinolytic therapy should be considered as therapy for neonatal renal vein thrombosis.

Purpose: A comparative cost analysis of the front-line acute lymphoblastic leukemia studies of two collaborative pediatric oncology groups was performed to document the charges incurred by the patients for only mandatory laboratory and radiographic data on each protocol.

Materials and methods: The required laboratory and radiographic data on each protocol were tabulated to derive the charges at Phoenix Children's Hospital in July 1992. A comparison between the two pediatric oncology groups was made for each risk category of patients with acute lymphoblastic leukemia.

Results: A consistent cost difference between the two groups was attributed to the relatively expensive laboratory support required on group Y protocols.

Conclusions: The changing reimbursement environment is affecting the practice patterns of pediatric oncologists, who must be cognizant of the charges for performing scientific clinical trials. Pediatric oncologists must continue to provide quality care without jeopardizing progress.

Purpose: We describe certain clinical, epidemiologic, and host-susceptibility features of Yersinia enterocolitica infection in the context of a patient with underlying risk factors.

Patients and methods: A 10-year-old black girl with sickle cell disease receiving chelation therapy for iron overload resulting from chronic transfusion therapy was admitted with acute abdominal pain and fever.

Results: Upon hospital admission, differential diagnoses included enterocolitis, appendicitis, and vasoocclusive crisis. On the 6th hospital day, the patient's stool culture became positive for Y. enterocolitica. Household exposure to raw pork intestines (chitterlings) was the presumed source of the infection. Deferoxamine therapy was withheld, and antibiotic therapy was administered with subsequent clinical improvement.

Conclusions: Y. enterocolitica infection should be considered as a cause of abdominal pain mimicking appendicitis in patients with underlying risk factors (including certain sickle cell patients). History of exposure to raw or undercooked pork products and appropriate cultures should be obtained. Deferoxamine therapy should be withheld in iron-overloaded patients presenting with such symptoms because deferoxamine and iron overload constitute independent risk factors for Yersinia infection. Such patients should be advised to avoid potential exposures to this pathogen.