Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review
Background: There is an association between treatment options and growth in patients with mucopolysaccharidoses (MPS). The appropriate management of MPS is an essential factor for the growth of the patients. Objectives: This study aims to review systematically the available data on the growth status...
| Main Authors: | , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Mazandaran University of Medical Sciences
2023-10-01
|
| Series: | Journal of Pediatrics Review |
| Subjects: | |
| Online Access: | http://jpr.mazums.ac.ir/article-1-534-en.pdf |
| _version_ | 1797352946858459136 |
|---|---|
| author | Seyed Ebrahim Tabatabayipoor Noushin Rostampour Silva Hovsepian Homyra Raispour Reza Hashemipour Arvin Khachikian Mahin Hashemipour |
| author_facet | Seyed Ebrahim Tabatabayipoor Noushin Rostampour Silva Hovsepian Homyra Raispour Reza Hashemipour Arvin Khachikian Mahin Hashemipour |
| author_sort | Seyed Ebrahim Tabatabayipoor |
| collection | DOAJ |
| description | Background: There is an association between treatment options and growth in patients with mucopolysaccharidoses (MPS). The appropriate management of MPS is an essential factor for the growth of the patients.
Objectives: This study aims to review systematically the available data on the growth status and related treatment factors in patients with MPS type I and MPS type II.
Methods: A systematic literature search was performed in PubMed, Scopus, and the Web of Science using related keywords by March 2023. In this systematic review, the primary outcome was determining the growth status (mainly height z-score) of patients with MPSI and MPSII from reviewed studies and its association with different treatment options. The author’s name, year of publication, country, type of MPS, growth status, treatment options, and any associations between growth status, disease, and treatment variables in the article were extracted.
Results: From the initially retrieved 743 references, 100 were removed due to being duplicates, 31 articles were evaluated by reading the full text, and finally 20 were included in the systematic review. Based on the analyses, treatment options improved growth in the MPS patients. Certain variables regarding the treatment were key factors, such as the age of treatment initiation, combination therapy, and human growth hormone therapy. Some factors related to the characteristics of the patients, including genotype (type of mutation) and disease severity, are also key factors. Patients with MPSI and MPSII had normal growth and height during the first years of life, but after 2-5 years, their growth rate decreases progressively.
Conclusions: The findings of this review indicated that growth impairment is common in patients with MPSI and MPSII. Treatment improved the growth development in these patients but not as much as expected. Some patients’ characteristics, such as disease severity and type of mutations, affect treatment efficacy and height gain. From treatment-related factors, the most important factor is the age of treatment initiation. Regarding other factors such as donor type, human growth hormone administration, and combination therapies, current findings are inconclusive, and more studies are needed. |
| first_indexed | 2024-03-08T13:24:09Z |
| format | Article |
| id | doaj.art-f5e2cbc91152451889bc9e63e6f28757 |
| institution | Directory Open Access Journal |
| issn | 2322-4398 2322-4401 |
| language | English |
| last_indexed | 2024-03-08T13:24:09Z |
| publishDate | 2023-10-01 |
| publisher | Mazandaran University of Medical Sciences |
| record_format | Article |
| series | Journal of Pediatrics Review |
| spelling | doaj.art-f5e2cbc91152451889bc9e63e6f287572024-01-17T15:45:15ZengMazandaran University of Medical SciencesJournal of Pediatrics Review2322-43982322-44012023-10-01114301314Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic ReviewSeyed Ebrahim Tabatabayipoor0Noushin Rostampour1Silva Hovsepian2Homyra Raispour3Reza Hashemipour4Arvin Khachikian5Mahin Hashemipour6 Metabolic Liver Disease Research Center, Isfahan University of Medical Sciences, Isfahan, Iran. Metabolic Liver Disease Research Center, Isfahan University of Medical Sciences, Isfahan, Iran. Metabolic Liver Disease Research Center, Isfahan University of Medical Sciences, Isfahan, Iran. Metabolic Liver Disease Research Center, Isfahan University of Medical Sciences, Isfahan, Iran. Department of Veterinary Medicine, Faculty of Veterinary, Karaj Branch, Islamic Azad University, Karaj, Iran. Department of Biological Sciences, School of Biological Sciences, University of California, Irvine, United States. Metabolic Liver Disease Research Center, Isfahan University of Medical Sciences, Isfahan, Iran. Background: There is an association between treatment options and growth in patients with mucopolysaccharidoses (MPS). The appropriate management of MPS is an essential factor for the growth of the patients. Objectives: This study aims to review systematically the available data on the growth status and related treatment factors in patients with MPS type I and MPS type II. Methods: A systematic literature search was performed in PubMed, Scopus, and the Web of Science using related keywords by March 2023. In this systematic review, the primary outcome was determining the growth status (mainly height z-score) of patients with MPSI and MPSII from reviewed studies and its association with different treatment options. The author’s name, year of publication, country, type of MPS, growth status, treatment options, and any associations between growth status, disease, and treatment variables in the article were extracted. Results: From the initially retrieved 743 references, 100 were removed due to being duplicates, 31 articles were evaluated by reading the full text, and finally 20 were included in the systematic review. Based on the analyses, treatment options improved growth in the MPS patients. Certain variables regarding the treatment were key factors, such as the age of treatment initiation, combination therapy, and human growth hormone therapy. Some factors related to the characteristics of the patients, including genotype (type of mutation) and disease severity, are also key factors. Patients with MPSI and MPSII had normal growth and height during the first years of life, but after 2-5 years, their growth rate decreases progressively. Conclusions: The findings of this review indicated that growth impairment is common in patients with MPSI and MPSII. Treatment improved the growth development in these patients but not as much as expected. Some patients’ characteristics, such as disease severity and type of mutations, affect treatment efficacy and height gain. From treatment-related factors, the most important factor is the age of treatment initiation. Regarding other factors such as donor type, human growth hormone administration, and combination therapies, current findings are inconclusive, and more studies are needed.http://jpr.mazums.ac.ir/article-1-534-en.pdfmucopolysaccharidosesgrowthtreatmentheight |
| spellingShingle | Seyed Ebrahim Tabatabayipoor Noushin Rostampour Silva Hovsepian Homyra Raispour Reza Hashemipour Arvin Khachikian Mahin Hashemipour Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review Journal of Pediatrics Review mucopolysaccharidoses growth treatment height |
| title | Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review |
| title_full | Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review |
| title_fullStr | Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review |
| title_full_unstemmed | Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review |
| title_short | Growth and Related Treatment Factors in Mucopolysaccharidoses Type I and II: A Systematic Review |
| title_sort | growth and related treatment factors in mucopolysaccharidoses type i and ii a systematic review |
| topic | mucopolysaccharidoses growth treatment height |
| url | http://jpr.mazums.ac.ir/article-1-534-en.pdf |
| work_keys_str_mv | AT seyedebrahimtabatabayipoor growthandrelatedtreatmentfactorsinmucopolysaccharidosestypeiandiiasystematicreview AT noushinrostampour growthandrelatedtreatmentfactorsinmucopolysaccharidosestypeiandiiasystematicreview AT silvahovsepian growthandrelatedtreatmentfactorsinmucopolysaccharidosestypeiandiiasystematicreview AT homyraraispour growthandrelatedtreatmentfactorsinmucopolysaccharidosestypeiandiiasystematicreview AT rezahashemipour growthandrelatedtreatmentfactorsinmucopolysaccharidosestypeiandiiasystematicreview AT arvinkhachikian growthandrelatedtreatmentfactorsinmucopolysaccharidosestypeiandiiasystematicreview AT mahinhashemipour growthandrelatedtreatmentfactorsinmucopolysaccharidosestypeiandiiasystematicreview |