Original Article

IJMDC. 2022; 6(6): 775-784

Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region

Authors: Sameh Awaad, Yahia Aktham, Marwan ElBagoury, Aly Ezzat, Sherif Roushdy.

ABSTRACT

Background: For the last few years, the diagnosis of lysosomal storage disorders (LSDs) has increased in Saudi Arabia and the Arabian Gulf region. Mucopolysaccharidosis (MPS) is one of the LSDs that showed high rates of consanguinity in Arabian countries. Therefore, an online survey was designed to measure the clinical knowledge of Saudi pediatricians about MPS I. Methods: A multinational, cross-sectional study was conducted to collect data through an online survey administered to pediatricians and other physicians mostly involved in MPS-I management. The survey was formed of two long clinical cases scenarios, seven close-ended questions related to general knowledge about MPS-I disease, and five close-ended questions related to MPS-I patient’s journey. Results: Study surveys were filled by 58 physicians. High rates of MPS diagnosis were noted among physicians’ responses. Other than musculoskeletal symptoms, physicians reported the following symptoms; hepato-splenomegaly, corneal clouding, skeletal abnormalities, coarse facies, hernias, and mental retardation. The multidisciplinary team was the most selected option when considering patients’ referrals. Regarding MPS complications, musculoskeletal abnormalities including irreversible skeletal deformities, growth retardation, and mobility loss were the most MPS late complications. Conclusion: The current results give key insights into the current status of clinical practice in countries of the Arabian Gulf region. Physicians of Kingdom of Saudi Arabia (KSA) and other countries of the Arabian Gulf region have solid knowledge and long experience in the management of MPS I patients.

Keywords:
Mucopolysaccharidosis, MPS, lysosomal storage disorders, Saudi Arabia, gulf.


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Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region


Authors
Sameh Awaad
Medical Affairs Department, Sanofi-Genzyme, Gulf region, Jeddah, Saudi Arabia
PubMed articlesGoogle scholar articles

Yahia Aktham
Medical Affairs Department, Sanofi-Genzyme, Gulf region, Jeddah, Saudi Arabia
PubMed articlesGoogle scholar articles

Marwan ElBagoury
Medical Affairs Department, Sanofi-Genzyme, Gulf region, Jeddah, Saudi Arabia
PubMed articlesGoogle scholar articles

Aly Ezzat
Medical Affairs Department, Sanofi-Genzyme, Gulf region, Jeddah, Saudi Arabia
PubMed articlesGoogle scholar articles

Sherif Roushdy
Medical Affairs Department, Sanofi-Genzyme, Gulf region, Jeddah, Saudi Arabia.
PubMed articlesGoogle scholar articles


Correspondence to:
. Sameh Awaad, Medical Affairs Department, Sanofi-Genzyme, Gulf region, Jeddah, Saudi Arabia.; sameh.awaad@sanofi.com

Publication history
Received 20 Sep 2021
Revised 20 Feb 2022
Accepted 16 Mar 2022
Published online 08 Apr 2022
Published in print 04 Jun 2022

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Pubmed Style

Awaad S, Aktham Y, ElBagoury M, Ezzat A, Roushdy S. Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region. IJMDC. 2022; 6(6): 775-784. doi:10.24911/IJMDC.51-1632095880


Web Style

Awaad S, Aktham Y, ElBagoury M, Ezzat A, Roushdy S. Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region. https://www.ijmdc.com/?mno=125831 [Access: August 18, 2022]. doi:10.24911/IJMDC.51-1632095880


AMA (American Medical Association) Style

Awaad S, Aktham Y, ElBagoury M, Ezzat A, Roushdy S. Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region. IJMDC. 2022; 6(6): 775-784. doi:10.24911/IJMDC.51-1632095880


Vancouver/ICMJE Style

Awaad S, Aktham Y, ElBagoury M, Ezzat A, Roushdy S. Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region. IJMDC. (2022), [cited August 18, 2022]; 6(6): 775-784. doi:10.24911/IJMDC.51-1632095880


Harvard Style

Awaad, S., Aktham, . Y., ElBagoury, . M., Ezzat, . A. & Roushdy, . S. (2022) Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region. IJMDC, 6 (6), 775-784. doi:10.24911/IJMDC.51-1632095880


Turabian Style

Awaad, Sameh, Yahia Aktham, Marwan ElBagoury, Aly Ezzat, and Sherif Roushdy. 2022. Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region. International Journal of Medicine in Developing Countries, 6 (6), 775-784. doi:10.24911/IJMDC.51-1632095880


Chicago Style

Awaad, Sameh, Yahia Aktham, Marwan ElBagoury, Aly Ezzat, and Sherif Roushdy. "Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region." International Journal of Medicine in Developing Countries 6 (2022), 775-784. doi:10.24911/IJMDC.51-1632095880


MLA (The Modern Language Association) Style

Awaad, Sameh, Yahia Aktham, Marwan ElBagoury, Aly Ezzat, and Sherif Roushdy. "Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region." International Journal of Medicine in Developing Countries 6.6 (2022), 775-784. Print. doi:10.24911/IJMDC.51-1632095880


APA (American Psychological Association) Style

Awaad, S., Aktham, . Y., ElBagoury, . M., Ezzat, . A. & Roushdy, . S. (2022) Mucopolysaccharidosis I: disease knowledge and patient’s journey in Saudi Arabia and Gulf Region. International Journal of Medicine in Developing Countries, 6 (6), 775-784. doi:10.24911/IJMDC.51-1632095880


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