Treatment of growth failure with growth hormone in children with chronic kidney disease

An open-label long-term study

D. E. Müller-Wiefel, H. Frisch, T. Tulassay, L. Bell, Z. Zadik

Research output: Contribution to journalArticle

6 Citations (Scopus)

Abstract

Aims: To assess long-term efficacy and safety of recombinant human growth hormone (GH) in childrenwith chronic kidney disease (CKD). Methods: An open-label, international, multicenter study. Children with CKD and growth failure received GH (0.35 mg/kg/week). The primary efficacy endpoint was a significant change in height velocity (HV) and height standard deviation score (SDS) versus baseline after 12months of treatment, extended to 24 months, then to 5 years. Results: In total, 81 patients enrolled (CKD Stage 4 - 5 = 37, on dialysis = 27, post-transplant = 17). After 12 and 24 months of treatment, increases were seen in mean (SD) HV (4.6 (3.1) to 9.0 (3.6) cm/year and 4.5 (3.3) to 7.5 (2.9) cm/year, respectively; both p <0.001), mean (SD) height SDS (-3.7 (1.7) to -3.0 (1.7) and -3.6 (1.5) to -2.5 (1.5), respectively; both p <0.001) and mean (SD) HV SDS (-2.4 (2.5) to 3.8 (4.5) and -2.4 (2.2) to 1.1 (3.8), respectively; both p <0.001). A normal height SDS was seen in 1% of children at baseline, 17%after 12months and 43% after 24months of treatment. Improvementswere similar across CKD subgroups with the greatest improvements in CKD Stage 4 - 5. Among 31 patients who completed about 5 years of treatment, four reached final height. There was no undue bone age acceleration and no deterioration of kidney function. Ten adverse events were related to GH treatment. Conclusions: In this long-term study, GH treatment was associated with significant improvements in growth and height in children with CKD and growth failure, and was well tolerated.

Original languageEnglish
Pages (from-to)97-105
Number of pages9
JournalClinical Nephrology
Volume74
Issue number2
Publication statusPublished - Aug 2010

Fingerprint

Treatment Failure
Chronic Renal Insufficiency
Growth Hormone
Growth
Therapeutics
Human Growth Hormone
Multicenter Studies
Dialysis
Transplants
Kidney
Safety
Bone and Bones

Keywords

  • Children
  • Chronic kidney disease
  • Clinical trial
  • Growth hormone treatment
  • Short stature

ASJC Scopus subject areas

  • Nephrology
  • Medicine(all)

Cite this

Treatment of growth failure with growth hormone in children with chronic kidney disease : An open-label long-term study. / Müller-Wiefel, D. E.; Frisch, H.; Tulassay, T.; Bell, L.; Zadik, Z.

In: Clinical Nephrology, Vol. 74, No. 2, 08.2010, p. 97-105.

Research output: Contribution to journalArticle

Müller-Wiefel, D. E. ; Frisch, H. ; Tulassay, T. ; Bell, L. ; Zadik, Z. / Treatment of growth failure with growth hormone in children with chronic kidney disease : An open-label long-term study. In: Clinical Nephrology. 2010 ; Vol. 74, No. 2. pp. 97-105.
@article{de03e8bb515646a59d57ffa80abb3b18,
title = "Treatment of growth failure with growth hormone in children with chronic kidney disease: An open-label long-term study",
abstract = "Aims: To assess long-term efficacy and safety of recombinant human growth hormone (GH) in childrenwith chronic kidney disease (CKD). Methods: An open-label, international, multicenter study. Children with CKD and growth failure received GH (0.35 mg/kg/week). The primary efficacy endpoint was a significant change in height velocity (HV) and height standard deviation score (SDS) versus baseline after 12months of treatment, extended to 24 months, then to 5 years. Results: In total, 81 patients enrolled (CKD Stage 4 - 5 = 37, on dialysis = 27, post-transplant = 17). After 12 and 24 months of treatment, increases were seen in mean (SD) HV (4.6 (3.1) to 9.0 (3.6) cm/year and 4.5 (3.3) to 7.5 (2.9) cm/year, respectively; both p <0.001), mean (SD) height SDS (-3.7 (1.7) to -3.0 (1.7) and -3.6 (1.5) to -2.5 (1.5), respectively; both p <0.001) and mean (SD) HV SDS (-2.4 (2.5) to 3.8 (4.5) and -2.4 (2.2) to 1.1 (3.8), respectively; both p <0.001). A normal height SDS was seen in 1{\%} of children at baseline, 17{\%}after 12months and 43{\%} after 24months of treatment. Improvementswere similar across CKD subgroups with the greatest improvements in CKD Stage 4 - 5. Among 31 patients who completed about 5 years of treatment, four reached final height. There was no undue bone age acceleration and no deterioration of kidney function. Ten adverse events were related to GH treatment. Conclusions: In this long-term study, GH treatment was associated with significant improvements in growth and height in children with CKD and growth failure, and was well tolerated.",
keywords = "Children, Chronic kidney disease, Clinical trial, Growth hormone treatment, Short stature",
author = "M{\"u}ller-Wiefel, {D. E.} and H. Frisch and T. Tulassay and L. Bell and Z. Zadik",
year = "2010",
month = "8",
language = "English",
volume = "74",
pages = "97--105",
journal = "Clinical Nephrology",
issn = "0301-0430",
publisher = "Dustri-Verlag Dr. Karl Feistle",
number = "2",

}

TY - JOUR

T1 - Treatment of growth failure with growth hormone in children with chronic kidney disease

T2 - An open-label long-term study

AU - Müller-Wiefel, D. E.

AU - Frisch, H.

AU - Tulassay, T.

AU - Bell, L.

AU - Zadik, Z.

PY - 2010/8

Y1 - 2010/8

N2 - Aims: To assess long-term efficacy and safety of recombinant human growth hormone (GH) in childrenwith chronic kidney disease (CKD). Methods: An open-label, international, multicenter study. Children with CKD and growth failure received GH (0.35 mg/kg/week). The primary efficacy endpoint was a significant change in height velocity (HV) and height standard deviation score (SDS) versus baseline after 12months of treatment, extended to 24 months, then to 5 years. Results: In total, 81 patients enrolled (CKD Stage 4 - 5 = 37, on dialysis = 27, post-transplant = 17). After 12 and 24 months of treatment, increases were seen in mean (SD) HV (4.6 (3.1) to 9.0 (3.6) cm/year and 4.5 (3.3) to 7.5 (2.9) cm/year, respectively; both p <0.001), mean (SD) height SDS (-3.7 (1.7) to -3.0 (1.7) and -3.6 (1.5) to -2.5 (1.5), respectively; both p <0.001) and mean (SD) HV SDS (-2.4 (2.5) to 3.8 (4.5) and -2.4 (2.2) to 1.1 (3.8), respectively; both p <0.001). A normal height SDS was seen in 1% of children at baseline, 17%after 12months and 43% after 24months of treatment. Improvementswere similar across CKD subgroups with the greatest improvements in CKD Stage 4 - 5. Among 31 patients who completed about 5 years of treatment, four reached final height. There was no undue bone age acceleration and no deterioration of kidney function. Ten adverse events were related to GH treatment. Conclusions: In this long-term study, GH treatment was associated with significant improvements in growth and height in children with CKD and growth failure, and was well tolerated.

AB - Aims: To assess long-term efficacy and safety of recombinant human growth hormone (GH) in childrenwith chronic kidney disease (CKD). Methods: An open-label, international, multicenter study. Children with CKD and growth failure received GH (0.35 mg/kg/week). The primary efficacy endpoint was a significant change in height velocity (HV) and height standard deviation score (SDS) versus baseline after 12months of treatment, extended to 24 months, then to 5 years. Results: In total, 81 patients enrolled (CKD Stage 4 - 5 = 37, on dialysis = 27, post-transplant = 17). After 12 and 24 months of treatment, increases were seen in mean (SD) HV (4.6 (3.1) to 9.0 (3.6) cm/year and 4.5 (3.3) to 7.5 (2.9) cm/year, respectively; both p <0.001), mean (SD) height SDS (-3.7 (1.7) to -3.0 (1.7) and -3.6 (1.5) to -2.5 (1.5), respectively; both p <0.001) and mean (SD) HV SDS (-2.4 (2.5) to 3.8 (4.5) and -2.4 (2.2) to 1.1 (3.8), respectively; both p <0.001). A normal height SDS was seen in 1% of children at baseline, 17%after 12months and 43% after 24months of treatment. Improvementswere similar across CKD subgroups with the greatest improvements in CKD Stage 4 - 5. Among 31 patients who completed about 5 years of treatment, four reached final height. There was no undue bone age acceleration and no deterioration of kidney function. Ten adverse events were related to GH treatment. Conclusions: In this long-term study, GH treatment was associated with significant improvements in growth and height in children with CKD and growth failure, and was well tolerated.

KW - Children

KW - Chronic kidney disease

KW - Clinical trial

KW - Growth hormone treatment

KW - Short stature

UR - http://www.scopus.com/inward/record.url?scp=77955764938&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=77955764938&partnerID=8YFLogxK

M3 - Article

VL - 74

SP - 97

EP - 105

JO - Clinical Nephrology

JF - Clinical Nephrology

SN - 0301-0430

IS - 2

ER -