Impact of maintenance treatment on male gonadal function in patients treated for localized rhabdomyosarcoma in RMS2005 trial in France.

Background: Maintenance treatment with vinorelbine and oral cyclophosphamide (oral-CPM) improves outcome of nonmetastatic high-risk (HR) and very-high risk (VHR) rhabdomyosarcoma (RMS) patients. However, gonadal toxicity of maintenance was not yet investigated.

Methods: The authors focused their analysis on male gonadal toxicity in HR/VHR groups of RMS2005 trial, in France.

Early adolescence assessment of eleven boys treated with continuous subcutaneous infusion (CSGI) of recombinant LH and FSH during infancy.

Introduction: We previously observed that continuous subcutaneous gonadotropin infusion (CSGI) in infants with congenital hypogonadotropic hypogonadism (CHH) can mimic minipuberty.

Objective: to describe the early adolescence outcome of boys treated during the first year of life.

Methods: In this retrospective cohort study, we describe 11 CHH boys aged 12 years [11.

Diagnosis and management of congenital hypopituitarism in children.

Hypopituitarism (or pituitary deficiency) is a rare disease with an estimated prevalence of between 1/16,000 and 1/26,000 individuals, defined by insufficient production of one or several anterior pituitary hormones (growth hormone [GH], thyroid-stimulating hormone [TSH], adrenocorticotropic hormone [ACTH], luteinizing hormone [LH], follicle-stimulating hormone [FSH], prolactin), in association or not with diabetes insipidus (antidiuretic hormone [ADH] deficiency). While in adults hypopituitarism is mostly an acquired disease (tumors, irradiation), in children it is most often a congenital condition, due to abnormal pituitary development. Clinical symptoms vary considerably from isolated to combined deficiencies and between syndromic and non-syndromic forms.

Risk factors for obesity in adulthood among survivors of childhood cancer.

Objective: The aim of this study was to identify risk factors for obesity in childhood cancer survivors (CCSs).

Methods: The study included 3199 patients of the French Childhood Cancer Survivor Study cohort, with 303 patients with obesity who had returned the self-questionnaire. Analyses were adjusted for social deprivation index and sex.

Long-term hospitalisations in survivors of paediatric solid tumours in France.

The late effects of treatments for childhood cancers may lead to severe and multiple health conditions requiring hospitalisation. We aimed to estimate the hospitalisation rate among childhood cancer survivors (CCS) in France, to compare them with the general population and to investigate the associated factors. We matched total of 5439 5-year solid CCS diagnosed before the age of 21 between 1945 and 2000 by sex, birth year and region of residence to 386,073 individuals of the French general population.

Health care expenditures among long-term survivors of pediatric solid tumors: Results from the French Childhood Cancer Survivor Study (FCCSS) and the French network of cancer registries (FRANCIM).

Background: Childhood cancer survivors (CCS) may require lifelong medical care due to late effects of cancer treatments. Little is known about of their healthcare utilization and expenditures at long-term especially in publicly funded health care system. We aim to estimate and describe the health care expenditures among long-term CCS in France.

Determinants of Final Height in Patients Born Small for Gestational Age Treated with Recombinant Growth Hormone.

Introduction: About 8% of children born small for gestational age (SGA) do not reach a final height within the normal range. Recombinant human growth hormone (rhGH) has been shown to be effective in increasing the final height in children born SGA. Our objective was to identify predictive factors of final height in children born SGA treated with rhGH.

Adrenocortical tumours in children and adolescents: The EXPeRT/PARTNER diagnostic and therapeutic recommendations.

Adrenocortical tumours (ACTs) are rare during childhood. A complete surgical resection provides the best chance of cure, but the role and efficacy of the adjuvant therapy are still controversial. Various histologic criteria of malignancy for ACTs adopted in children do not facilitate comparative studies and are not completely shared.

An eHealth Framework for Managing Pediatric Growth Disorders and Growth Hormone Therapy.

Background: The use of technology to support health and health care has grown rapidly in the last decade across all ages and medical specialties. Newly developed eHealth tools are being implemented in long-term management of growth failure in children, a low prevalence pediatric endocrine disorder.

Objective: Our objective was to create a framework that can guide future implementation and research on the use of eHealth tools to support patients with growth disorders who require growth hormone therapy.

Influence of growth hormone therapy on the occurrence of a second neoplasm in survivors of childhood cancer.

Context: Growth hormone (GH) deficiency is a common late effect of cranial irradiation. However, concerns have been raised that GH treatment might lead to an increased risk of a second neoplasm (SN).

Objective: To study the impact of GH treatment on the risk of SN in a French cohort of survivors of childhood cancer (CCS) treated before 1986.

Risk Factors for Small Adult Height in Childhood Cancer Survivors.

Purpose: Between 10% and 20% of childhood cancer survivors (CCS) experience impaired growth, leading to small adult height (SAH). Our study aimed to quantify risk factors for SAH or growth hormone deficiency among CCS.

Methods: The French CCS Study holds data on 7,670 cancer survivors treated before 2001.

Exploring heterogeneity of adrenal cortical tumors in children: The French pediatric rare tumor group (Fracture) experience.

Introduction: Pediatric adrenal cortical tumors are characterized by a wide spectrum of behavior. Questions remain regarding intermediate disease stages with isolated tumor rupture or relapse.

Objectives: To describe clinical characteristics, treatment strategy, and outcome of patients depending on disease stage, tumor rupture, or in case of a refractory tumor, to discuss optimal management.

Revisiting the role of the pathological grading in pediatric adrenal cortical tumors: results from a national cohort study with pathological review.

The prognosis of malignant pediatric adrenocortical tumors is closely related to disease stage, which is used to guide perioperative treatment recommendations. However, current scoring systems are inadequate to distinguish between benign and malignant adrenocortical tumors. Robust microscopic prognostic features that could help determine perioperative therapy are also lacking.

Cerebrovascular Diseases in Childhood Cancer Survivors: Role of the Radiation Dose to Willis Circle Arteries.

Background And Purpose: The aim of this study was to investigate the role of radiation dose received to the circle of Willis (WC) during radiation therapy (RT) and of potential dose-response modifiers on the risk of stroke after treatment of childhood cancer.

Methods: We evaluated the risk factors for stroke in a cohort of 3172 5-year survivors of childhood cancer who were followed up for a median time of 26 years. Radiation doses to the WC and brain structures were estimated for each of the 2202 children who received RT.

Report of two novel mutations in PTHLH associated with brachydactyly type E and literature review.

Autosomal-dominant brachydactyly type E is a congenital limb malformation characterized by small hands and feet as a result of shortened metacarpals and metatarsals. Alterations that predict haploinsufficiency of PTHLH, the gene coding for parathyroid hormone related protein (PTHrP), have been identified as a cause of this disorder in seven families. Here, we report three patients affected with brachydactyly type E, caused by PTHLH mutations expected to result in haploinsufficiency, and discuss our data compared to published reports.

Dose-Effect Relationship of Alkylating Agents on Testicular Function in Male Survivors of Childhood Lymphoma.

The purpose of our study was to assess the gonadal function in male survivors of childhood lymphoma. We studied 171 male survivors of childhood lymphoma (83 with B-cell non-Hodgkin lymphoma [B-NHL], 32 with T-cell non-Hodgkin lymphoma [T-NHL], 50 with Hodgkin lymphoma [HL], and 6 with anaplastic large-cell lymphoma [ALCL]), measuring follicle-stimulating hormone [FSH] and luteinizing hormone [LH] levels at a median age of 21.1 (17-30.

[Endocrine sequelae after treatment of pediatric cancer: From childhood to adulthood].

Endocrine sequelae are among the most frequently reported complications in childhood cancer survivors, affecting 40 to 60% of these patients during adult life. Most of these complications are the result of cranial radiation therapy for brain or facial tumor, lymphoma or leukemia. The present review describes the main endocrine disturbances observed in this population, including disorders of hypothalamic-pituitary axis, especially the frequently observed growth hormone deficiency and disorders of puberty, thyroid and parathyroid dysfunction, obesity and metabolic syndrome, alterations in glucose metabolism and decreased bone mineral density.

[Gonadal function after treatment for a childhood or adolescent cancer].

Due to high cure rate in childhood and adolescent cancer, fertility preservation is a major concern. Chemotherapy, radiotherapy and surgery may alter gonadal function, and uterine cavity in women. In women, combined toxicity affecting both endocrine function and ovulation are observed leading to premature ovarian insufficiency.

Ovarian reserve after treatment with alkylating agents during childhood.

Study Question: What is the effect of different alkylating agents used without pelvic radiation to treat childhood cancer in girls on the ovarian reserve in survivors?

Summary Answer: Ovarian reserve seems to be particularly reduced in survivors who received procarbazine (in most cases for Hodgkin lymphoma) or high-dose chemotherapy; procarbazine but not cyclophosphamide dose is associated with diminished ovarian reserve.

What Is Known Already: A few studies have demonstrated diminished ovarian reserve in survivors after various combination therapies, but the individual role of each treatment is difficult to assess.

Study Design: Prospective cross-sectional study, involving 105 survivors and 20 controls.

[Fertility preservation in children and teenagers with cancer].

Given the excellent survival rate of children and teenagers treated for cancer, careful should be given in order to minimize long-term late effects of treatments and to improve quality of life. In this regard, fertility preservation is particularly important. Age, sex and concomitant therapies have an impact on the level of risk of hypofertility, with a stronger risk for chemotherapeutic regimens including alkylating agents, and gonads or hypothalamo-pituitary axis radiotherapy.