Philippe F Backeljauw

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States

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Publications (20)44.83 Total impact

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    ABSTRACT: Blood flow in human arteries has been investigated using computational fluid dynamics tools. This paper considers flow modeling through three aorta models reconstructed from cross-sectional magnetic resonance scans of female patients. One has the normal control configuration, the second has elongation of the transverse aorta, and the third has tortuosity of the aorta with stenosis. The objective of this study is to determine the impact of aortic abnormal geometries on the wall shear stress (WSS), luminal surface low-density lipoproteins (LDLs) concentration, and oxygen flux along the arterial wall. The results show that the curvature of the aortic arch and the stenosis have significant effects on the blood flow, and in turn, the mass transport. The location of hypoxia areas can be predicted well by ignoring the effect of hemoglobin on the oxygen transport. However, this simplification indeed alters the absolute value of Sherwood number on the wall.
    Journal of biomechanics 10/2013; · 2.66 Impact Factor
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    ABSTRACT: Background: Treatment with recombinant human insulin-like growth factor-I (IGF-I) stimulates linear growth in children with severe IGF-I deficiency (IGFD). Aims: To evaluate the efficacy and safety of treatment with IGF-I in patients with severe IGFD treated until adult or near-adult height. Methods: Twenty-one children with severe IGFD were treated until adult or near-adult height under a predominantly open-label design. All patients were naive to IGF-I. Recombinant human IGF-I was administered subcutaneously in doses between 60 and 120 µg/kg twice daily. Nine patients received additional therapy with gonadotropin- releasing hormone (GnRH) analog for a mean period of 2.9 ± 1.8 years. Results: Mean duration of treatment was 10.0 years. Mean height velocity increased from 3.1 cm/year prior to treatment to 7.4 cm/year during the first year of treatment. Height velocities during the subsequent years were lower, but remained above baseline for up to 12 years. Cumulative mean Δ height SD score at (near) adult height was +2. The observed mean gain in height was 13.4 cm more than had been expected without treatment. The adult height achieved by the patients also treated with GnRH analog was not different from those who received IGF-I therapy alone. There were no new safety signals identified in these patients, a subset of those previously reported. Conclusion: Long-term therapy with IGF-I improves adult height of patients with severe IGFD. Most patients did not bring their heights into the normal adult range.
    Hormone Research in Paediatrics 07/2013; · 1.55 Impact Factor
  • Iris Gutmark-Little, Philippe F Backeljauw
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    ABSTRACT: Girls and women with Turner syndrome (TS) have a highly increased morbidity as the result of cardiovascular disease, both congenital and acquired. Increased clinical use of cardiac magnetic resonance imaging (CMR) in TS patients over recent years has allowed for characterization of disease not always possible with standard imaging modalities, such as echocardiography (echo). In this review, we discuss the current literature regarding CMR in TS patients and guidelines for its use. © 2013 Blackwell Publishing Ltd.
    Clinical Endocrinology 01/2013; · 3.40 Impact Factor
  • Philippe F Backeljauw, Steven D Chernausek
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    ABSTRACT: Specific lesions of the growth hormone (GH)/insulin-like growth factor (IGF) axis have been identified in humans, each of which has distinctive auxologic and biochemical features. Measures of circulating IGF-I are useful in diagnosing growth disorders in childhood and in evaluating response to GH therapy. Recombinant human IGF-I is an effective treatment of severe primary IGF deficiency, which is typical of patients with GH receptor defects (Laron syndrome). Such treatment has been limited to a few severely affected patients. Future studies will provide new insight into IGF-I as treatment and into the nature of growth disorders that involve the IGF axis.
    Endocrinology and metabolism clinics of North America 06/2012; 41(2):265-82, v. · 3.56 Impact Factor
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    ABSTRACT: Growth hormone (GH) is widely prescribed for children with short stature across a range of growth disorders. Recombinant human (rh) insulin-like growth factor-1 (rhIGF-1) therapy is approved for severe primary IGF-I deficiency - a state of severe GH resistance. Evidence is increasing for an unacceptably high rate of poor or unsatisfactory response to growth-promoting therapy (i.e. not leading to significant catch up growth) in terms of change in height standard deviation score (SDS) and height velocity (HV) in many approved indications. Consequently, there is a need to define poor response and to prevent or correct it by optimizing treatment regimens within accepted guidelines. Recognition of a poor response is an indication for action by the treating physician, either to modify the therapy or to review the primary diagnosis leading either to discontinuation or change of therapy. This review discusses the optimal investigation of the child who is a candidate for GH or IGF-1 therapy so that a diagnosis-based choice of therapy and dosage can be made. The relevant parameters in the evaluation of growth response are described together with the definitions of poor response. Prevention of poor response is addressed by discussion of strategy for first-year management with GH and IGF-1. Adherence to therapy is reviewed as is the recommended action following the identification of the poorly responding patient. The awareness, recognition and management of poor response to growth-promoting therapy will lead to better patient care, greater cost-effectiveness and increased opportunities for clinical benefit.
    Clinical Endocrinology 04/2012; 77(2):169-81. · 3.40 Impact Factor
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    ABSTRACT: Cardiovascular disease affects >50% of Turner syndrome (TS) patients. With newer imaging modalities, this prevalence increases and the spectrum of recognized anomalies broadens. To determine the prevalence and hemodynamic significance of partial anomalous pulmonary venous return (PAPVR) in adolescents and young adults with TS using transthoracic echocardiogram (TTE) and cardiac magnetic resonance (CMR), and to study the association with phenotype. The records of 39 young TS patients who had received TTE and CMR were reviewed. PAPVR was diagnosed in seven (18%) patients; six were newly diagnosed by CMR after normal TTE. In one subject, PAPVR was associated with right ventricular enlargement and a pulmonic blood flow (Qp) to systemic blood flow (Qs) ratio of 1.9:1.0, necessitating surgical repair. In other subjects with and without PAPVR, Qp:Qs and the right ventricle to left ventricle end-diastolic volume ratio were statistically different. Other clinical features were not predictive of PAPVR. The prevalence of PAPVR is high in TS, and it may be hemodynamically significant. Diagnosis by TTE can be difficult. Appropriate screening and management are indicated.
    Journal of pediatric endocrinology & metabolism: JPEM 01/2012; 25(5-6):435-40. · 0.75 Impact Factor
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    ABSTRACT: Turner syndrome affects one in 2,500 girls and women and is associated with cardiovascular anomalies. Visualizing the descending thoracic aorta in adults with Turner syndrome with echocardiography is difficult. Therefore, cardiac MRI is the preferred imaging modality for surveillance. Our goals were to use cardiac MRI describe the spectrum and frequency of cardiovascular abnormalities and to evaluate aortic dilatation and associated abnormalities in pediatric patients with Turner syndrome. The cases of 51 patients with Turner syndrome (median age, 18.4 years; range, 6-36 years) were evaluated with cardiac MRI. The characteristics assessed included aortic structure, elongation of the transverse aortic arch, aortic diameter at multiple locations, and coarctation of the aorta (CoA). Additional evaluations were made for presence of bicuspid aortic valve (BAV), and partial anomalous pulmonary venous return (PAPVR). Associations between the cardiac MRI data and the following factors were assessed: age, karyotype, body surface area, blood pressure, and ventricular sizes and function. Sixteen patients (31.4%) had elongation of the transverse aortic arch, eight (15.7%) had CoA, 20 (39.2%) had BAV, and eight (15.7%) had PAPVR. Aortic dilatation was most common at the aortic sinus (30%). Elongation of the transverse aortic arch was associated with CoA (p < 0.01) and BAV (p < 0.05). Patients with elongation of the transverse aortic arch had dilated aortic sinus (p < 0.05). Patients with PAPVR had increased right heart mass (p < 0.05), increased ratio of main pulmonary artery to aortic valve blood flow (p = 0.0014), and increased right ventricular volume (p < 0.05). Cardiovascular anomalies in pediatric patients with Turner syndrome include aortic abnormalities and PAPVR. The significant association between elongation of the transverse aortic arch and CoA, BAV, and aortic sinus dilatation may contribute to increased risk of aortic dissection. The presence of PAPVR can be hemodynamically significant. These findings indicate that periodic cardiac MRI screening of persons with Turner syndrome is beneficial.
    American Journal of Roentgenology 02/2011; 196(2):454-60. · 2.90 Impact Factor
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    ABSTRACT: To identify the incidence of endocrine dysfunction in children following traumatic brain injury (TBI). This was a prospective evaluation of 31 children after TBI. Inclusion criteria included Glasgow Coma Scale score ≤ 12 and age 1.5-18 years. We evaluated thyroid function, insulin-like growth factor I, insulin-like growth factor-binding protein 3, and cortisol at 1, 3, 6, and 12 months after injury, and assessed prolactin at 3 and 6 months. At 6 months, we also assessed overnight spontaneous growth hormone secretion, nocturnal thyrotropin surge, adrenal reserve, and serum and urine osmolarity. The average patient age was 11.6 years, and mean Glascow Coma Scale score was 6. The incidence of endocrine dysfunction was 15% at 1 month, 75% at 6 months, and 29% at 12 months. At 12 months after injury, 14% had precocious puberty, 9% had hypothyroidism, and 5% had growth hormone deficiency. Endocrine dysfunction at 1 year did not correlate with the severity of injury. Endocrine dysfunction after TBI is common in children, but most cases resolve by 1 year. We recommend endocrine surveillance at both 6 and 12 months following moderate or severe TBI to ensure early intervention for persistent or late-occurring endocrine sequelae.
    The Journal of pediatrics 12/2010; 157(6):894-9. · 4.02 Impact Factor
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    ABSTRACT: With the availability of recombinant human (rh) IGF-I as a new therapeutic agent, the criteria for diagnosis and strategies for management of growth deficiencies continue to evolve. This supplement provides a clinical update on molecular, therapeutic, and metabolic aspects of the management of short stature associated with insulin-like growth factor-I deficiency (IGFD). Several distinct, single-gene defects associated with primary IGFD now have been identified. The first section of this supplement focuses on selecting certain patients for specific genetic testing of the GH/IGF-I axis, based on previously obtained clinical and biochemical assessments. Management of short stature in children responding poorly to rhGH and definitions of a good and poor response are discussed in the next section. In addition, the authors further address different methods to help practicing clinicians predict and assess GH response, review the effect of rhGH on final adult height, and discuss the role IGF-I may have in the therapeutic approach to short stature. Finally, the metabolic aspects related to the treatment of short stature are discussed in the third part of this supplement.
    Pediatric endocrinology reviews: PER 02/2010; 7 Suppl 1:154-71.
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    ABSTRACT: To determine the prevalence of adrenal insufficiency in infants with hemangiomas following treatment with systemic glucocorticoids (GCs). Prospective study for 18 months. Hemangioma and vascular malformation center at a tertiary care children's hospital. Sixteen infants with hemangiomas had an adrenal axis evaluation as soon as possible following the completion of GC therapy. Ten healthy control infants were also evaluated for comparison. Prednisolone at a starting dose of 2 to 3 mg/kg/d for 4 weeks, followed by a tapering period. The mean duration of GC treatment was 7.2 months. Prevalence of adrenal insufficiency in GC-treated subjects as assessed by a combination low-dose/high-dose corticotropin stimulation test. Subjects underwent corticotropin testing at a mean of 13 days after the completion of therapy. Only 1 of the 16 GC-treated infants (6%) had adrenal insufficiency. This subject was tested 1 day after GC treatment was stopped, and results from retesting 3 months later were normal. All control subjects had normal adrenal function. Infants with hemangiomas are at low risk of adrenal insufficiency following the completion of GC therapy, as used in our hemangioma center.
    Archives of dermatology 04/2009; 145(3):262-6. · 4.76 Impact Factor
  • Philippe Backeljauw
    Pediatric endocrinology reviews: PER 05/2008; 5 Suppl 3:853-6.
  • Pediatric endocrinology reviews: PER 06/2007; 4 Suppl 2:252-71.
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    ABSTRACT: Children with severe IGF-I deficiency due to congenital or acquired defects in GH action have short stature that cannot be remedied by GH treatment. The objective of the study was to examine the long-term efficacy and safety of recombinant human IGF-I (rhIGF-I) therapy for short children with severe IGF-I deficiency. Seventy-six children with IGF-I deficiency due to GH insensitivity were treated with rhIGF-I for up to 12 yr under a predominantly open-label design. The study was conducted at general clinical research centers and with collaborating endocrinologists. Entry criteria included: age older than 2 yr, sd scores for height and circulating IGF-I concentration less than -2 for age and sex, and evidence of resistance to GH. rhIGF-I was administered sc in doses between 60 and 120 microg/kg twice daily. Height velocity, skeletal maturation, and adverse events were measured. Height velocity increased from 2.8 cm/yr on average at baseline to 8.0 cm/yr during the first year of treatment (P < 0.0001) and was dependent on the dose administered. Height velocities were lower during subsequent years but remained above baseline for up to 8 yr. The most common adverse event was hypoglycemia, which was observed both before and during therapy. It was reported by 49% of treated subjects. The next most common adverse events were injection site lipohypertrophy (32%) and tonsillar/adenoidal hypertrophy (22%). Treatment with rhIGF-I stimulates linear growth in children with severe IGF-I deficiency due to GH insensitivity. Adverse events are common but are rarely of sufficient severity to interrupt or modify treatment.
    Journal of Clinical Endocrinology &amp Metabolism 03/2007; 92(3):902-10. · 6.43 Impact Factor
  • Philippe F Backeljauw, Steven D Chernausek
    Hormone Research 02/2006; 65 Suppl 1:21-7. · 2.48 Impact Factor
  • Jefferson P Lomenick, Philippe F Backeljauw, Anne W Lucky
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    ABSTRACT: Hemangiomas, common proliferative vascular tumors, can grow rapidly in the first months of life. Although therapy with high-dose oral glucocorticoids is standard for lesions that threaten vital functions or are disfiguring, little is known about the endocrine consequences of this treatment. Using retrospective data, we examined growth velocity, changes in bone mineral density, and adrenal function in infants with hemangiomas treated with systemic glucocorticoids. Treatment consisted of oral prednisolone 2 to 4 mg/kg/day or dexamethasone 1 mg/kg/day. Mean growth velocity Z score on glucocorticoid therapy was -1.41 standard deviations in 13 patients. In four infants with adequate follow-up, growth velocity increased to +1.90 standard deviations after glucocorticoid treatment (Delta growth velocity +3.31 standard deviations). Mean lumbar spine bone mineral density Z score was -2.46 standard deviations before glucocorticoid treatment and -1.08 standard deviations at the end of treatment in six infants. Adrenal function after glucocorticoid therapy was assessed by low-dose adrenocorticotropic hormone stimulation test in 10 infants. Eight had a normal cortisol response, and one had a borderline response. One infant, who had been treated with dexamethasone, had an abnormal test result. In conclusion, systemic glucocorticoid treatment in infants with hemangiomas slowed linear growth, but "catch-up" growth was observed after treatment ceased. Glucocorticoids did not affect bone mineralization adversely. Only 1 of 10 glucocorticoid-treated infants had clear evidence of adrenal insufficiency after therapy was stopped.
    Pediatric Dermatology 01/2006; 23(2):169-74. · 1.04 Impact Factor
  • Meilan M Rutter, Sampath Prahalad, Murray Passo, Philippe F Backeljauw
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    ABSTRACT: A 15 year-old boy presented with moderate hypercalcemia (serum calcium 3.35 mmol/l) and eosinophilic fasciitis. An extensive search for a cause of hypercalcemia or underlying malignancy proved negative. Glucocorticoid treatment resulted in significant clinical improvement and resolution of the hypercalcemia. This report describes the novel association of hypercalcemia with eosinophilic fasciitis, with potential implications for the role of inflammatory mediators.
    Journal of pediatric endocrinology & metabolism: JPEM 10/2004; 17(9):1251-4. · 0.75 Impact Factor
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    Jefferson P Lomenick, Wendy A Jackson, Philippe F Backeljauw
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    ABSTRACT: Amiodarone is an iodine-rich drug used to treat cardiac dysrhythmias. The structure of amiodarone resembles that of thyroxine, and treatment with amiodarone may alter thyroid function. The effects of antenatal amiodarone use on fetal/neonatal thyroid function have only been addressed in a limited number of patient reports. We describe two cases of transient neonatal hypothyroidism due to in utero amiodarone exposure, followed by a brief review of the available literature.
    Journal of Perinatology 07/2004; 24(6):397-9. · 2.25 Impact Factor
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    Philippe F. Backeljauw, Susan R. Rose, Margaret Lawson
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    ABSTRACT: We review the main concerns related to the management of menstruation in adolescent females with developmental delay. Results from a questionnaire evaluating the current practice of menstrual control in developmentally delayed females by pediatric endocrinologists are also summarized. Guidelines for clinical care are provided, taking into account the specific health issues of this particular patient population.
    The Endocrinologist 02/2004; 14(2):87-92. · 0.12 Impact Factor
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    Journal of Pediatrics 10/2003; 143(4):415-21. · 4.04 Impact Factor
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    ABSTRACT: Deficiency of insulin-like growth factor-I (IGF-I) results in growth failure. A variety of molecular defects have been found to underlie severe primary IGF-I deficiency (IGFD), in which serum IGF-I concentrations are substantially decreased and fail to respond to GH therapy. Identification of more patients with primary or secondary IGFD is likely with investigative and diagnostic progress, particularly in the assessment of children with idiopathic short stature. Diagnosis of IGFD requires accurate and reliable IGF-I assays, adequate normative data for reference, and knowledge of IGF-I physiology for proper interpretation of data. Recombinant human IGF-I (rhIGF-I) treatment improves stature in patients with severe primary IGFD, and has also been shown to improve glycaemic control and insulin sensitivity in patients with severe insulin resistance. Ongoing studies of patients receiving rhIGF-I will allow further evaluation of the clinical utility of this treatment, with concurrent increase in our understanding of IGF-I and conditions of IGFD.
    Journal of pediatric endocrinology & metabolism: JPEM 23(1-2):3-16. · 0.75 Impact Factor