Nocturnal enuresis in pediatric sickle cell disease.

To assess the prevalence of nocturnal enuresis in children and adolescents with sickle cell disease (SCD) and associated factors, structured telephone interviews were conducted with primary caregivers of 217 children and adolescents with SCD aged 5 years or older. Prevalence, perceived causes, interventions undertaken, and emotional impact were assessed. Nocturnal enuresis was significantly higher for males (28.2% of males) than for females (11% of females), p = .002, and compared with cited population prevalence rates, nocturnal enuresis was significantly higher for children with SCD, p < .01. SCD was the most common reason given by primary caregivers for enuresis. Primary caregivers used a wide range of interventions for nocturnal enuresis, but few used empirically supported treatments for enuresis or spoke with their health care team about the enuresis. These data suggest that systematic assessment and intervention for nocturnal enuresis must be implemented in the follow-up care of children and adolescents with SCD.

Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease type 1b.

The purpose of this study was to evaluate the efficacy and toxicity of recombinant human granulocyte colony-stimulating factor (rhG-CSF) therapy in patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease (GSD) type 1b. Thirteen patients with neutropenia and/or neutrophil dysfunction secondary to GSD type 1b were treated with rhG-CSF. The effects of therapy on neutrophil numbers and in vitro neutrophil function and on bone marrow cellularity and morphology were studied. The clinical status of the patients and the occurrence of adverse events associated with rhG-CSF use were monitored. Use of rhG-CSF therapy was associated with a significant increase in circulating neutrophil numbers (P <. 01) and an improvement in neutrophil function as assessed in vitro. In addition, rhG-CSF therapy produced a significant increase in marrow cellularity and an increase in myeloid:erythroid (M:E) ratio, indicating stimulation of granulopoeisis. No adverse effects on marrow function were noted; in particular, no myelodysplasia or marrow exhaustion was seen. Use of rhG-CSF therapy was associated with objective and subjective improvements in infection-related morbidity. The therapy was well tolerated, although all patients developed splenomegaly, and 5 patients developed mild hypersplenism that did not require any specific treatment. rhG-CSF therapy is efficacious in the management of neutropenia and neutrophil dysfunction associated with GSD type 1b. Patients on this therapy need to be monitored for hypersplenism. Continued follow-up will be necessary to confirm long-term safety; however, no significant short-term toxicity was noted.

Severe iron deficiency anemia in young children.

We retrospectively characterized clinical features of 55 patients with severe nutritional iron deficiency anemia. Anemia was commonly discovered in the absence of related complaints. Forty percent of patients were of Southeast Asian ancestry. Most were treated successfully with iron therapy alone; 8 required transfusion.

Use of hydroxyurea in children with sickle cell disease: what comes next?

Hydroxyurea (HU) is the first drug that, under well-organized clinical trials, has shown the potential for altering significantly the clinical severity of sickle cell disease (SCD). The placebo-controlled trial of HU in adult SS hemoglobinopathy patients (the Multicenter Study of Hydroxyurea in Sickle Cell Anemia) reported in May 1995 that HU therapy reduced significantly the frequencies of severe pain episodes, acute chest syndrome, and transfusion. Despite these impressive results, no guidelines have been developed to direct clinicians on the use of HU in adult SCD patients. Small-scale phase II studies in children have reported increases in fetal hemoglobin (HbF) and F-cell levels in response to HU therapy. A larger phase II study, the Pediatric Hydroxyurea Study Group (HUG-KIDS), is under way and is expected to be completed by March 1998. The need for a large-scale placebo-controlled trial in children will be doubtful if no unusual short-term toxicity is demonstrated by HUG-KIDS. Guidelines regarding patient selection, dosing schedules, treatment goals, and short- and long-term monitoring parameters need to be established. The case is made of the organization of a clinical network to register and follow SCD patients treated with HU for long-term toxicity.

Pneumococcal colonization in children with sickle cell disease [see comment].

OBJECTIVE: The goals of this prospective study were to define the Streptococcus pneumoniae colonization rate in children with sickle cell disease (SCD) at the Children's Hospital of Philadelphia and to determine the serotype and antibiotic susceptibility of all isolates. METHODS: Children with SCD followed at the hospital were sampled for colonization with S. pneumoniae by means of a throat or nasopharyngeal swab on one or two occasions. Patient information was obtained when the specimen was collected. Specimens were isolated on gentamicin-blood agar plates and modified Avery broth. Antibiotic susceptibility was determined by a commercially available test (E-test). Isolates were serotyped with the use of type-specific antisera. The relationship between the data noted above and certain clinical parameters was examined. RESULTS: A total of 490 specimens were obtained from 278 patients. Twenty-eight patients had a culture positive for S. pneumoniae, resulting in an overall colonization rate of 10%. Thirty-three percent (11/33) of all isolates were resistant to penicillin-seven intermediately resistant and four highly resistant. Twelve percent of isolates were also resistant to cefotaxime. Eight different serotypes were identified; all but one are included in the current 23-valent pneumococcal vaccine. Penicillin prophylaxis did not increase the rate of colonization with resistant strains of pneumococcus. CONCLUSION: Our results do not support a change in the current use of penicillin prophylaxis nor in the acute management of the febrile child with SCD.