학술논문
Growth hormone treatment for children with mucopolysaccharidosis I or II
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- 영문명
- Growth hormone treatment for children with mucopolysaccharidosis I or II
- 발행기관
- 대한의학유전학회
- 저자명
- Minji Im Chiwoo Kim Juyoung Sung Insung Kim Ji-Hoon Hwang Min-Sun Kim Sung Yoon Cho
- 간행물 정보
- 『대한의학유전학회지』제20권 제2호, 60~69쪽, 전체 10쪽
- 주제분류
- 의약학 > 기타의약학
- 파일형태
- 발행일자
- 2023.12.31
4,000원
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국문 초록
영문 초록
Purpose: Despite enzyme replacement therapy (ERT) and/or allogeneic hematopoietic stem cell transplantation, individuals with mucopolysaccharidosis (MPS) I or II often experience significant growth deficiencies. This study aimed to assess the safety and efficacy of recombinant human growth hormone (hGH) treatment in children diagnosed with MPS I or II.
Materials and Methods: A total of nine pediatric patients—four with MPS I and five with MPS II—underwent treatment with ERT and hGH at Samsung Medical Center.
Results: The mean hGH dose administered was 0.26±0.03 mg/kg/week. In the MPS I group, three patients showed an increase in height Z-score from -4.09±0.83 to -3.68±0.43 after 1 year of hGH treatment, and to -3.10±0.72 by the end of the hGH regimen. In the MPS II group, while the height Z-score of four patients decreased according to standard growth charts, it improved from 1.61±1.79 to 2.71±1.68 based on the disease-specific growth chart through hGH treatment. Two patients discontinued hGH treatment due to lack of efficacy after 22 and 6 months each of treatment, respectively. No new-onset neurological symptoms or necessity for prosthetic or orthopedic surgery were reported during hGH treatment.
Conclusion: This study provides insights into the impact of hGH on MPS patients, demonstrating its potential to reverse growth deceleration in some cases. Further research is needed to explore the long-term effects of hGH on changes in body composition, muscle strength, and bone health in this population.
목차
Introduction
Materials and Methods
Results
Discussion
Acknowledgements
Funding
Authors' Contributions
References
해당간행물 수록 논문
- Exome and genome sequencing for diagnosing patients with suspected rare genetic disease
- Recent progress in using Drosophila as a platform for human genetic disease research
- A Korean case of CTCF related neurodevelopmental disorders
- Growth hormone treatment for children with mucopolysaccharidosis I or II
- Exonic copy number variations in rare genetic disorders
- Differential microbiota network according to colorectal cancer lymph node metastasis stages
참고문헌
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