Impact of Conventional Medical Therapy on Bone Mineral Density and Bone Turnover in Adult Patients with X-Linked Hypophosphatemia: A 6-Year Prospective Cohort Study

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Abstract

X-linked hypophosphatemia (XLH) is a rare, inheritable disorder manifesting as rickets in children and osteomalacia in adults. While conventional medical treatment with oral phosphate and alfacalcidol is recommended in childhood, it is undecided whether adults should continue therapy. The aim of this 6-year prospective study was to determine the impact of conventional medical treatment on areal bone mineral density (aBMD), bone turnover markers (BTMs) and measures of calcium homeostasis in 27 adult patients with XLH, 11 of whom received medical treatment. Lumbar spine and total hip aBMD, as assessed by DXA, and biochemical measures of calcium, phosphate, PTH, 1,25 dihydroxyvitamin D2+3 (1,25(OH)2D), fibroblast growth factor 23 (FGF23), P1NP and CTX were measured at baseline and at follow-up. The renal tubular reabsorption of PO4 (TmPO4/GFR) was calculated at both time points. Multilevel mixed-effects linear regression models were used for analyses. During the study period, spine and hip aBMD did not change significantly between treated and non-treated XLH patients. There was a trend towards a decrease in calcium, phosphate and TmPO4/GFR in the treatment group (p = 0.057, p = 0.080 and p = 0.063, respectively), whereas PTH, FGF23, 1,25(OH)2D and P1NP did not change significantly in either groups. However, CTX increased significantly in the treated compared to non-treated group (p = 0.044). Continuing conventional medical therapy in adulthood, although associated with increased bone resorption, does not promote or prevent loss of bone mass as evidenced from the stable aBMD of the hip and spine in XLH patients.

Original languageEnglish
JournalCalcified Tissue International
Volume102
Issue number3
Pages (from-to)321–328
ISSN0171-967X
DOIs
Publication statusPublished - Mar 2018

Fingerprint

Familial Hypophosphatemic Rickets
Bone Density
Cohort Studies
Prospective Studies
Linear Models
Fibroblast Growth Factor 1
Osteomalacia
Hip
Homeostasis

Keywords

  • Adolescent
  • Adult
  • Aged
  • Bone Density Conservation Agents/therapeutic use
  • Bone Density/drug effects
  • Bone Remodeling/drug effects
  • Familial Hypophosphatemic Rickets/drug therapy
  • Female
  • Fibroblast Growth Factors/metabolism
  • Humans
  • Lumbar Vertebrae/drug effects
  • Male
  • Middle Aged
  • Prospective Studies
  • Young Adult

Cite this

@article{6303f4690baf45e0b89a29d556c1bea9,
title = "Impact of Conventional Medical Therapy on Bone Mineral Density and Bone Turnover in Adult Patients with X-Linked Hypophosphatemia: A 6-Year Prospective Cohort Study",
abstract = "X-linked hypophosphatemia (XLH) is a rare, inheritable disorder manifesting as rickets in children and osteomalacia in adults. While conventional medical treatment with oral phosphate and alfacalcidol is recommended in childhood, it is undecided whether adults should continue therapy. The aim of this 6-year prospective study was to determine the impact of conventional medical treatment on areal bone mineral density (aBMD), bone turnover markers (BTMs) and measures of calcium homeostasis in 27 adult patients with XLH, 11 of whom received medical treatment. Lumbar spine and total hip aBMD, as assessed by DXA, and biochemical measures of calcium, phosphate, PTH, 1,25 dihydroxyvitamin D2+3 (1,25(OH)2D), fibroblast growth factor 23 (FGF23), P1NP and CTX were measured at baseline and at follow-up. The renal tubular reabsorption of PO4 (TmPO4/GFR) was calculated at both time points. Multilevel mixed-effects linear regression models were used for analyses. During the study period, spine and hip aBMD did not change significantly between treated and non-treated XLH patients. There was a trend towards a decrease in calcium, phosphate and TmPO4/GFR in the treatment group (p = 0.057, p = 0.080 and p = 0.063, respectively), whereas PTH, FGF23, 1,25(OH)2D and P1NP did not change significantly in either groups. However, CTX increased significantly in the treated compared to non-treated group (p = 0.044). Continuing conventional medical therapy in adulthood, although associated with increased bone resorption, does not promote or prevent loss of bone mass as evidenced from the stable aBMD of the hip and spine in XLH patients.",
keywords = "Adolescent, Adult, Aged, Bone Density Conservation Agents/therapeutic use, Bone Density/drug effects, Bone Remodeling/drug effects, Familial Hypophosphatemic Rickets/drug therapy, Female, Fibroblast Growth Factors/metabolism, Humans, Lumbar Vertebrae/drug effects, Male, Middle Aged, Prospective Studies, Young Adult",
author = "Shanbhogue, {Vikram Vinod} and Stinus Hansen and J{\o}rgensen, {Niklas Rye} and Beck-Nielsen, {Signe Sparre}",
year = "2018",
month = "3",
doi = "10.1007/s00223-017-0363-3",
language = "English",
volume = "102",
pages = "321–328",
journal = "Calcified Tissue International",
issn = "0171-967X",
publisher = "Springer",
number = "3",

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TY - JOUR

T1 - Impact of Conventional Medical Therapy on Bone Mineral Density and Bone Turnover in Adult Patients with X-Linked Hypophosphatemia

T2 - A 6-Year Prospective Cohort Study

AU - Shanbhogue, Vikram Vinod

AU - Hansen, Stinus

AU - Jørgensen, Niklas Rye

AU - Beck-Nielsen, Signe Sparre

PY - 2018/3

Y1 - 2018/3

N2 - X-linked hypophosphatemia (XLH) is a rare, inheritable disorder manifesting as rickets in children and osteomalacia in adults. While conventional medical treatment with oral phosphate and alfacalcidol is recommended in childhood, it is undecided whether adults should continue therapy. The aim of this 6-year prospective study was to determine the impact of conventional medical treatment on areal bone mineral density (aBMD), bone turnover markers (BTMs) and measures of calcium homeostasis in 27 adult patients with XLH, 11 of whom received medical treatment. Lumbar spine and total hip aBMD, as assessed by DXA, and biochemical measures of calcium, phosphate, PTH, 1,25 dihydroxyvitamin D2+3 (1,25(OH)2D), fibroblast growth factor 23 (FGF23), P1NP and CTX were measured at baseline and at follow-up. The renal tubular reabsorption of PO4 (TmPO4/GFR) was calculated at both time points. Multilevel mixed-effects linear regression models were used for analyses. During the study period, spine and hip aBMD did not change significantly between treated and non-treated XLH patients. There was a trend towards a decrease in calcium, phosphate and TmPO4/GFR in the treatment group (p = 0.057, p = 0.080 and p = 0.063, respectively), whereas PTH, FGF23, 1,25(OH)2D and P1NP did not change significantly in either groups. However, CTX increased significantly in the treated compared to non-treated group (p = 0.044). Continuing conventional medical therapy in adulthood, although associated with increased bone resorption, does not promote or prevent loss of bone mass as evidenced from the stable aBMD of the hip and spine in XLH patients.

AB - X-linked hypophosphatemia (XLH) is a rare, inheritable disorder manifesting as rickets in children and osteomalacia in adults. While conventional medical treatment with oral phosphate and alfacalcidol is recommended in childhood, it is undecided whether adults should continue therapy. The aim of this 6-year prospective study was to determine the impact of conventional medical treatment on areal bone mineral density (aBMD), bone turnover markers (BTMs) and measures of calcium homeostasis in 27 adult patients with XLH, 11 of whom received medical treatment. Lumbar spine and total hip aBMD, as assessed by DXA, and biochemical measures of calcium, phosphate, PTH, 1,25 dihydroxyvitamin D2+3 (1,25(OH)2D), fibroblast growth factor 23 (FGF23), P1NP and CTX were measured at baseline and at follow-up. The renal tubular reabsorption of PO4 (TmPO4/GFR) was calculated at both time points. Multilevel mixed-effects linear regression models were used for analyses. During the study period, spine and hip aBMD did not change significantly between treated and non-treated XLH patients. There was a trend towards a decrease in calcium, phosphate and TmPO4/GFR in the treatment group (p = 0.057, p = 0.080 and p = 0.063, respectively), whereas PTH, FGF23, 1,25(OH)2D and P1NP did not change significantly in either groups. However, CTX increased significantly in the treated compared to non-treated group (p = 0.044). Continuing conventional medical therapy in adulthood, although associated with increased bone resorption, does not promote or prevent loss of bone mass as evidenced from the stable aBMD of the hip and spine in XLH patients.

KW - Adolescent

KW - Adult

KW - Aged

KW - Bone Density Conservation Agents/therapeutic use

KW - Bone Density/drug effects

KW - Bone Remodeling/drug effects

KW - Familial Hypophosphatemic Rickets/drug therapy

KW - Female

KW - Fibroblast Growth Factors/metabolism

KW - Humans

KW - Lumbar Vertebrae/drug effects

KW - Male

KW - Middle Aged

KW - Prospective Studies

KW - Young Adult

U2 - 10.1007/s00223-017-0363-3

DO - 10.1007/s00223-017-0363-3

M3 - Journal article

C2 - 29143140

VL - 102

SP - 321

EP - 328

JO - Calcified Tissue International

JF - Calcified Tissue International

SN - 0171-967X

IS - 3

ER -