A trial of growth hormone therapy in well‐controlled hypophosphataemic rickets

Cameron,; Sochett,; Daneman,; Kooh,

doi:10.1046/j.1365-2265.1999.00680.x

Cited by 19 publications

(21 citation statements)

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“…[9][10][11][12][13][14][15] The patients were treated for periods of 0.5 to 9 years, with rhGH doses of 0.15 to 0.56 mg/kg per week. The median increase in standardized height amounted to 1.2 SD (range: 0.7-1.4).…”

Section: E594mentioning

confidence: 99%

“…8 Previous studies demonstrated that treatment with recombinant human growth hormone (rhGH) was able to improve short-and long-term longitudinal growth among small children with XLH. [9][10][11][12][13][14][15] However, we and others 11,12 noted that short-term rhGH treatment could aggravate disproportionate growth among these children. Here we report the effects of long-term rhGH treatment on longitudinal growth and body proportions among 3 initially prepubertal, short children with XLH who were prospectively monitored after the initiation of rhGH treatment, until their final heights were achieved.…”

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Effects of Growth Hormone Treatment on Body Proportions and Final Height Among Small Children With X-Linked Hypophosphatemic Rickets

et al. 2004

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ABSTRACT. Background. X-linked hypophosphatemic rickets (XLH) is characterized by rickets, disproportionate short stature, and impaired renal phosphate reabsorption and vitamin D metabolism. Despite oral phosphate and vitamin D treatment, most children with XLH demonstrate reduced adult height.Objective. To determine the beneficial effects of recombinant human growth hormone (rhGH) therapy on body proportions and adult height among patients with XLH.Methods. Three initially prepubertal short children (age, 9.4 -12.9 years) with XLH were treated with rhGH for 3.1 to 6.3 years until adult height was attained.Results. rhGH treatment led to sustained increases in standardized height for all children. The median adult height was 0.9 SD (range: 0.5-1.3 SD) greater than that at the initiation of rhGH treatment and exceeded the predicted adult height by 6.2 cm (range: 5.3-9.8 cm). However, longitudinal growth of the trunk was stimulated more than leg growth. During rhGH treatment, the standardized sitting height increased by 1.6 SD (range: 1.1-2.7 SD), compared with baseline values. In contrast, the median subischial leg length did not change consistently (median change: 0.3 SD; range: ؊0.1 to 0.6 SD).Conclusion. The increase in final height after rhGH treatment is of potential benefit for children with XLH. However, the exaggeration of disproportionate truncal growth observed for our prepubertal patients is a potential negative effect of treatment and should be confirmed with additional studies. Pediatrics 2004;113:e593-e596. URL: http://www.pediatrics.org/cgi/content/full/113/ 6/e593; growth hormone treatment, hypophosphatemic rickets, growth failure, disproportionate growth, final height.ABBREVIATIONS. XLH, X-linked hypophosphatemic rickets; GH, growth hormone; rhGH, recombinant human growth hormone. X -linked hypophosphatemic rickets (XLH) is an inherited disorder of phosphate homeostasis characterized by disproportionate short stature, rickets and osteomalacia, hypophosphatemia, aberrant phosphate reabsorption, and disturbance of vitamin D metabolism. 1 XLH is caused by mutations in the PHEX gene, encoding a membrane-bound endopeptidase. PHEX is expressed in bones and teeth but not in kidney, and efforts are underway to elucidate how PHEX function relates to the mutant phenotype. 2

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Section: E594mentioning

confidence: 99%

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Effects of Growth Hormone Treatment on Body Proportions and Final Height Among Small Children With X-Linked Hypophosphatemic Rickets

et al. 2004

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“…On the other hand, GH therapy resulted in an increase in bone mineral density and in linear growth at the long term [17, 18, 19]. In a recent study [20], however, adjunctive GH therapy did not offer benefit in linear growth or rachitic disease markers in well-controlled HR patients.…”

Section: Discussionmentioning

confidence: 99%

“…Based on these findings, GH has been used in combination with conventional therapy in HR in several studies which reported a better metabolic control at the short term [12, 13, 14, 15, 16, 17]and increased growth rate and bone mineral density at the long term [16, 17, 18, 19]after combined therapy over a period of 6–72 months. However, in a recent study performed by Cameron et al [20], adjunctive GH therapy did not offer any benefit in the management of HR over 12 months.…”

Section: Introductionmentioning

confidence: 99%

The Effect of Growth Hormone Treatment on Biochemical Indices in Hypophosphatemic Rickets

et al. 2001

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Background: Growth hormone (GH) in combination with conventional therapy in hypophosphatemic rickets (HR) has been shown to promote renal phosphate (P) conservation and to result in a better metabolic control. This study aimed at investigating the acute biochemical effects of GH in 7 patients (5 female, 2 male) with HR aged between 2.16 and 16 years. Methods: Each patient received the following in a sequential design: oral P plus 1,25-dihydroxyvitamin D₃ [1,25-(OH)₂D₃] therapy to determine the optimum doses for baseline requirement followed by GH-only therapy and GH +1,25-(OH)₂D₃ therapy and GH + P +1,25-(OH)₂D₃ therapy each for 2 weeks with 1 washout week off treatment in between. GH was given at a dose of 0.03 mg/kg/day s.c. on a daily basis. The dose of oral P used ranged between 500 and 2,000 mg/day, and the dose of 1,25-(OH)₂D₃ ranged between 0.25 and 0.5 µg/day and was kept constant for each child throughout the study. Results: Laboratory investigations repeated at the end of each treatment, and the first washout period showed that the serum P level was highest (2.9 ng/ml) during the GH + P + 1,25-(OH)₂D₃ period with higher serum 1,25-(OH)₂D₃ levels: 50.9 ± (SD) 23.4 ng/l. Parathyroid hormone and alkaline phosphatase levels did not show a significant difference between the periods. The tubular P reabsorbtion rate showed an insignificant increase during GH therapy periods. Conclusion: Considering the fixed dose of P and calcitriol, it may be concluded that GH added to conventional treatment in HR resulted in a slight improvement in the biochemical parameters without any side effects at the short term.

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“…The results have varied in terms of reestablishment of phosphorus homeostasis and the achievement of a better growth performance (62)(63)(64)(65). In two studies improvement in growth was noticed but with increased disproportion between upper and lower segments, predominating the trunk growth (66,67).…”

Section: Treatmentmentioning

confidence: 99%

Hypophosphatemic rickets and osteomalacia

Filho

Castro²,

Damiani

2006

Arq Bras Endocrinol Metab

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The hypophosphatemic conditions that interfere in bone mineralization comprise many hereditary or acquired diseases, all of them sharing the same pathophysiologic mechanism: reduction in the phosphate reabsorption by the renal tubuli. This process leads to chronic hyperphosphaturia and hypophosphatemia, associated with inappropriately normal or low levels of calcitriol, causing osteomalacia or rickets in children and osteomalacia in adults. X-linked hypophosphatemic rickets, autosomaldominant hypophosphatemic rickets, and tumor-induced osteomalacia are the main syndromes involved in the hypophosphatemic rickets. Although these conditions exhibit different etiologies, there is a common link among them: increased activity of a phosphaturic factor, being the fibroblast growth factor 23 (FGF-23) the most studied one and to which is attributed a central role in the pathophysiology of the hyperphosphaturic disturbances. Activating mutations of FGF-23 and inactivating mutations in the PHEX gene (a gene on the X chromosome that codes for a Zn-metaloendopeptidase proteolytic enzyme which regulates the phosphate) involved in the regulation of FGF-23 have been identified and have been implicated in the pathogenesis of these disturbances. Genetic studies tend to show that the phosphorus homeostasis depends on a complex osteo-renal metabolic axis, whose mechanisms of interaction have been poorly understood so far. This paper reviews the current knowledge status concerning the pathophysiology of phosphate metabolism regulation and the pathophysiologic basis of hypophosphatemic rickets. It also analyzes the clinical picture and the therapeutic aspects of these conditions as well. RESUMORaquitismo Hipofosfatêmico e Osteomalácia. Os distúrbios hipofosfatêmicos que comprometem a mineralização óssea englobam várias doenças, hereditárias e adquiridas, as quais compartilham um mesmo mecanismo fisiopatológico: a diminuição da reabsorção de fosfato nos túbulos renais. Este processo promove hiperfosfatúria e hipofosfatemia crônicas, associadas a níveis inapropriadamente normais ou baixos de 1,25 (OH) 2 D 3 , com conseqüente desordem do metabolismo ósteo-mineral, resultando em raquitismo e osteomalá-cia na faixa etária pediátrica e em osteomalácia nos adultos. O raquitismo hipofosfatêmico ligado ao X, o raquitismo hipofosfatêmico autossômico dominante e a osteomalácia induzida por tumor são as principais síndromes que constituem os raquitismos hipofosfatêmicos. Apesar de estas doenças apresentarem etiopatogenias distintas, as evidências bioquímico-moleculares indicam uma base fisiopatológica em comum: maior atividade de um agente fosfatúrico, sendo o fator de crescimento do fibroblasto 23 (FGF-23) o mais estudado e ao qual é atribuído um papel central na fisiopatologia destes distúrbios. Várias mutações ativadoras do gene do FGF-23 e mutações inativadoras do

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A trial of growth hormone therapy in well‐controlled hypophosphataemic rickets

Abstract: In well-controlled hypophosphataemic rickets patients receiving conventional therapy, adjunctive therapy with standard dose rhGH offers no benefits in linear growth or rachitic disease markers.

Cited by 19 publications

References 21 publications

Effects of Growth Hormone Treatment on Body Proportions and Final Height Among Small Children With X-Linked Hypophosphatemic Rickets

Effects of Growth Hormone Treatment on Body Proportions and Final Height Among Small Children With X-Linked Hypophosphatemic Rickets

The Effect of Growth Hormone Treatment on Biochemical Indices in Hypophosphatemic Rickets

Hypophosphatemic rickets and osteomalacia

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