Energy expenditure in obesity associated with craniopharyngioma.

Background And Purpose: Obesity is a common yet incompletely understood complication of childhood craniopharyngioma. We hypothesized that craniopharyngioma is associated with specific defects in energy balance compared to obese control children.

Methods: Eleven craniopharyngioma patients were recruited for a study on body composition and energy balance.

Pediatric thyroid-associated orbitopathy: the Children's Hospital of Philadelphia experience and literature review.

Background: As Graves' disease is uncommon in children, Graves' eye disease should be even more unusual. Here we report our experience with Graves' eye disease at the Children's Hospital of Philadelphia and review the literature on ophthalmic findings in children with Graves' disease.

Summary: A retrospective review identified 152 children with Graves' disease seen in the endocrinology clinic of the Children's Hospital of Philadelphia over a 3-year period.

Late effects in survivors of tandem peripheral blood stem cell transplant for high-risk neuroblastoma.

Background: Increasing numbers of children with advanced neuroblastoma are achieving cure. We describe the clinical late effects specific to survivors of stage IV neuroblastoma all similarly treated using tandem autologous peripheral blood stem cell rescue with TBI.

Method: The medical records of 35 neuroblastoma patients treated at CHOP between 1997 and 2001 were examined.

Increased prevalence of ADHD in Turner syndrome with no evidence of imprinting effects.

Background: Turner syndrome (TS) results from the loss of part or all of one X chromosome in females. It can result in short stature, various dysmorphic findings, and difficulties with psychosocial adjustment. Girls with TS have previously been found to exhibit increased levels of hyperactivity and inattention.

Use of the recombinant human TSH stimulated thyroglobulin level and diagnostic whole body scan in children with differentiated thyroid carcinoma.

The recombinant human thyrotropin (TSH) (rhTSH) stimulated thyroglobulin (Tg) level is a useful tumor marker for disease surveillance in adults with differentiated thyroid carcinoma (DTC). We report our institution's experience using rhTSH in children. Seven children with DTC on thyroid hormone suppressive therapy after total thyroidectomy and radioablation received rhTSH (0.

Vasopressin and desmopressin in central diabetes insipidus: adverse effects and clinical considerations.

The management of central diabetes insipidus has been greatly simplified by the introduction of desmopressin (DDAVP). Its ease of administration, safety and tolerability make DDAVP the first line agent for outpatient treatment of central diabetes insipidus. The major complication of DDAVP therapy is water intoxication and hyponatremia.

Salutary effects of combining early very low-dose systemic estradiol with growth hormone therapy in girls with Turner syndrome.

Context: Optimizing pubertal estrogen replacement in girls with Turner syndrome is important.

Objective: The study objective was to test the hypotheses that physiological estradiol replacement administered early with GH will preserve height potential as much as if administered late and that it will bring about a greater height gain than standard oral estrogen therapy combined with GH.

Design: The study was randomized to early or late estrogen treatment; follow-up was at 3.

Multiple endocrine neoplasia type 2A in a kindred with C634Y mutation.

Multiple endocrine neoplasia type 2A (MEN 2A) is most frequently caused by codon 634 activating mutations. Medullary thyroid carcinoma has occurred before the age of 2, with pheochromocytomas and primary hyperparathyroidism occurring later in childhood. We report cases of 4 siblings with C634Y-positive MEN 2A (all <11 years old): 3 with medullary thyroid carcinoma (1 had nodal metastasis, and another had a parathyroid adenoma) and 1 with C-cell hyperplasia.

Growth hormone releasing hormone plus arginine stimulation testing in young adults treated in childhood with cranio-spinal radiation therapy.

Objective: Growth hormone deficiency (GHD) secondary to cranio-spinal radiation therapy (CSRT) is a complication seen in medulloblastoma survivors. The standard for diagnosis of adult GHD is a peak GH < 3 microg/l by the insulin tolerance test (ITT). However, insulin tolerance testing exposes patients to the risks of hypoglycaemia.

Adult heights attained by children with hypothalamic/chiasmatic glioma treated with growth hormone.

Hypothalamic/chiasmatic gliomas (H/CG) in children are commonly accompanied by endocrine dysfunction due to mass effects of the tumor itself or as a consequence of tumor therapy, with GH deficiency (GHD) being the most common disorder. We report the height outcomes of GH-treated H/CG patients with GHD. We reviewed the records of 14 GHD patients with H/CG who were treated with human GH.

Is treatment with growth hormone effective in children with cerebral palsy?

Children with cerebral palsy (CP) often have poor linear growth during childhood, resulting in a diminished final adult height. Here we report a female with CP and short stature but without growth hormone (GH) deficiency who exhibited increased growth during treatment with GH. We also report two other children with CP who were treated with GH: one female with a history of leukemia, and a male with Klinefelter syndrome.

Endocrine outcome in children with medulloblastoma treated with 18 Gy of craniospinal radiation therapy.

Craniospinal radiation therapy (CSRT) combined with chemotherapy results in significant endocrine morbidity. Between 1987 and 1990, a trial using 18 Gy was conducted to treat 10 young children with medulloblastoma. There were 7 survivors.

Surgery with or without radiation therapy in the management of craniopharyngiomas in children and young adults.

Purpose: The optimal management of craniopharyngiomas remains controversial, especially in children and young adults. This study reports a single institution's experience with such patients.

Methods And Materials: Between 1974 and 2001, 76 patients were treated for craniopharyngioma at the Children's Hospital of Philadelphia and the Hospital of University of Pennsylvania (HUP).

Prolactin levels during long-term risperidone treatment in children and adolescents.

Background: This analysis was designed to investigate prolactin levels in children and adolescents on long-term risperidone treatment and explore any relationship with side effects hypothetically attributable to prolactin (SHAP).

Method: Data from 5 clinical trials (total N = 700) were pooled for this post hoc analysis. Children and adolescents aged 5 to 15 years with subaverage intelligence quotients and conduct or other disruptive behavior disorders received risperidone treatment (0.

Adult height and adult sitting height in childhood medulloblastoma survivors.

Outcome in adult height and sitting height is poor in children surviving medulloblastoma due to craniospinal irradiation (CSRT) and chemotherapy. We evaluated adult height and sitting height in 51 medulloblastoma patients stratified into four groups: G1, GH-deficient (GHD) patients treated with 23-39 Gy CSRT but not treated with GH [recombinant human (rh)GH]; G2, patients treated with rhGH; G3, patients who were not GHD; and G4, patients treated with 18 Gy CSRT and rhGH. Standing/sitting height of each group was compared with parental height and previously reported outcome studies.

Effect of growth hormone (GH) treatment on bone in postpubertal GH-deficient patients: a 2-year randomized, controlled, dose-ranging study.

GH treatment in children with GH deficiency is frequently terminated at final height. However, in healthy individuals bone mass continues to accrue until peak bone mass is achieved. Because no prospective data specifically prove the role of GH in attainment of peak bone mass, we performed a multinational, controlled, 2-yr study in patients who had terminated pediatric GH at final height.

Long-term (15 years) outcome in an infant with metastatic adrenocortical carcinoma.

Adrenocortical carcinoma is a rare malignancy in children, with a high mortality. Little is known about long-term outcome, especially in infants treated with mitotane. We report the successful long-term outcome of a case of metastatic adrenocortical carcinoma presenting in infancy treated with surgical resection and mitotane.

Expanded spectrum of recombinant human growth hormone therapy.

The efficacy of recombinant human growth hormone in the treatment of growth hormone deficiency is well established. In recent years, the use of recombinant human growth hormone as a therapeutic modality has greatly increased and has expanded beyond the realm of replacement for growth hormone deficiency. Recombinant human growth hormone has been employed to ameliorate growth failure in multiple other disorders.

A multicenter study of the efficacy and safety of sustained release GH in the treatment of naive pediatric patients with GH deficiency.

Treatment of naive children with GH deficiency has relied upon long-term replacement therapy with daily injections of GH. The daily schedule may be inconvenient for patients and their caregivers, possibly promoting nonadherence with the treatment regimen or premature termination of treatment. We studied a new sustained release GH formulation, administered once or twice monthly, to determine its efficacy and safety in this population.

Prednicarbate emollient cream 0.1% in pediatric patients with atopic dermatitis.

Atopic dermatitis, common among infants and children, is an intensely pruritic, chronic, inflammatory dermatosis that is traditionally treated with emollients for dry skin and topical corticosteroids for inflamed areas. A multicenter, 3-week, open-label study evaluated prednicarbate emollient cream 0.1%, a nonhalogenated midpotency corticosteroid, in 55 patients aged 4 months to 12 years who were diagnosed with atopic dermatitis.

Characterization of the murine Lbx2 promoter, identification of the human homologue, and evaluation as a candidate for Alström syndrome.

The murine Lbx2 gene is a member of the ladybird family of homeobox genes, which is expressed in the developing urogenital system, eye, and brain. Using transgenic mice, we demonstrate that 9 kb of the 5' flanking region of mouse Lbx2 is able to direct expression of a reporter gene in a tissue-specific manner recapitulating the endogenous expression pattern. This regulatory region provides a novel reagent allowing for transgenic expression in the developing urogenital ridge.

Hyponatremia and polyuria in children with central diabetes insipidus: challenges in diagnosis and management.

Five patients with well-controlled, long-standing, central diabetes insipidus had acute development of dehydration, hyponatremia, and inappropriate natriuresis in the setting of polyuria resistant to exogenous antidiuretic hormone. Hyponatremia and dehydration worsened with fluid restriction or use of exogenous antidiuretic hormone. We discuss the challenges in diagnosis and management of probable salt wasting in children with central diabetes insipidus.

Growth hormone replacement therapy in children with medulloblastoma: use and effect on tumor control.

Purpose: Progress has been made in the treatment of medulloblastoma, the most common childhood malignant brain tumor: However, many long-term survivors will have posttherapy growth hormone insufficiency with resultant linear growth retardation. Growth hormone replacement therapy (GHRT) may significantly improve growth, but there is often reluctance to initiate GHRT because of concerns of an increased likelihood of tumor relapse.

Patients And Methods: This study retrospectively reviewed the use of GHRT for survivors of medulloblastoma in 11 neuro-oncology centers in North America who received initial treatment for disease between 1980 and 1993 to determine its impact on disease control.

Somatic growth failure after the Fontan operation.

Our study was designed to characterize the patterns of growth, in the medium term, of children with functionally univentricular hearts managed with a hemi-Fontan procedure in infancy, followed by a modified Fontan operation in early childhood. Failure of growth is common in patients with congenital cardiac malformations, and may be related to congestive heart failure and hypoxia. Repair of simple lesions appears to reverse the retardation in growth.