Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data

Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data Publisher Pubmed

Ashrafi MR^{1, 2} ; Babaee M³ ; Hashemi Nazari SS⁴ ; Barzegar M⁵ ; Ghazavi M⁶ ; Beiraghi Toosi M⁷ ; Nafissi S⁸ ; Inaloo S⁹ ; Zamani Ghaletaki G¹ ; Fatehi F⁸ ; Heshmat R¹⁰ ; Ghahvechi Akbari M¹¹ ; Abdi A¹² ; Bakhtiary H¹³ Show All Authors

Ashrafi MR^{1, 2}
Babaee M³
Hashemi Nazari SS⁴
Barzegar M⁵
Ghazavi M⁶
Beiraghi Toosi M⁷
Nafissi S⁸
Inaloo S⁹
Zamani Ghaletaki G¹
Fatehi F⁸
Heshmat R¹⁰
Ghahvechi Akbari M¹¹
Abdi A¹²
Bakhtiary H¹³
Montazerlotfelahi H¹⁴
Abbaskhanian A¹⁵
Hosseini SA¹⁶
Farshadmoghadam H¹⁷
Hosseiny SMM¹⁸
Shariatmadari F¹⁹
Ziaadini B²⁰
Babaei M²¹
Tavasoli A²²
Nikbakht S²³
Momen A²⁴
Khajeh A²⁵
Aminzadeh V²⁶
Mollamohammadi M²⁷
Taghdiri MM²⁸
Nasehi MM²⁸
Memarian S¹
Badv RS¹
Heidari M¹
Jafari N²⁸

Source: Journal of neuromuscular diseases Published:2024

Abstract

BACKGROUND: Three medications have been approved for spinal muscular atrophy (SMA) treatment. No head-to-head clinical trials have directly compared the efficacy of nusinersen and risdiplam. We compare the efficacy of them in Type 2 and 3 SMA patients, with 6 months of follow-up. METHODS: A multicenter cohort study was conducted. Demographic, genetic and clinical findings containing Hammersmith Functional Motor Scale Expanded (HFMSE) and revised upper limb module (RULM) scales were gathered. An increase of at least 3 points in HFMSE and RULM scores was considered a positive response. RESULTS: 73 (58.4%) children received risdiplam, and 52 (41.6%) received nusinersen non-randomly, based on clinical decision. The difference in HFMSE and RULM scores compared to the baseline was significant in both groups (P-value <0.001). However, there was no significant difference between mean difference changes in HFMSE and RULM scores between the two groups. 80.4% of patients in the risdiplam group and 72% in the nusinersen group achieved the 3-point cutoff after 6 months, and there is no significant difference between the two groups (P-Value:0.33). CONCLUSIONS: This study showed that both medications significantly changed the HFSME and RULM after 3 and 6 months of follow-up. However, there was no significant difference between the two drugs according to the HFSME.

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Experts (# of related papers)

Omid Yaghini (1)

Style	Citing Format
MLA	Ashrafi MR, et al.. "Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data." Journal of neuromuscular diseases, vol. 11, no. 6, 2024, pp. 1190-1199.
APA	Ashrafi MR, Babaee M, Hashemi Nazari SS, Barzegar M, Ghazavi M, Beiraghi Toosi M, Nafissi S, Inaloo S, Zamani Ghaletaki G, Fatehi F, Heshmat R, Ghahvechi Akbari M, Abdi A, Bakhtiary H, Montazerlotfelahi H, Abbaskhanian A, Hosseini SA, Farshadmoghadam H, Hosseiny SMM, ... Jafari N (2024). Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data. Journal of neuromuscular diseases, 11(6), 1190-1199.
Chicago	Ashrafi MR, Babaee M, Hashemi Nazari SS, Barzegar M, Ghazavi M, Beiraghi Toosi M, Nafissi S, et al.. "Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data." Journal of neuromuscular diseases 11, no. 6 (2024): 1190-1199.
Harvard	Ashrafi MR et al. (2024) 'Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data', Journal of neuromuscular diseases, 11(6), pp. 1190-1199.
Vancouver	Ashrafi MR, Babaee M, Hashemi Nazari SS, Barzegar M, Ghazavi M, Beiraghi Toosi M, et al.. Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data. Journal of neuromuscular diseases. 2024;11(6):1190-1199.
BibTex	@article{ author = {Ashrafi MR and Babaee M and Hashemi Nazari SS and Barzegar M and Ghazavi M and Beiraghi Toosi M and Nafissi S and Inaloo S and Zamani Ghaletaki G and Fatehi F and Heshmat R and Ghahvechi Akbari M and Abdi A and Bakhtiary H and Montazerlotfelahi H and Abbaskhanian A and Hosseini SA and Farshadmoghadam H and Hosseiny SMM and Shariatmadari F and Ziaadini B and Babaei M and Tavasoli A and Nikbakht S and Momen A and Khajeh A and Aminzadeh V and Mollamohammadi M and Taghdiri MM and Nasehi MM and Memarian S and Badv RS and Heidari M and Jafari N}, title = {Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data}, journal = {Journal of neuromuscular diseases}, volume = {11}, number = {6}, pages = {1190-1199}, year = {2024} }
RIS	TY - JOUR AU - Ashrafi MR AU - Babaee M AU - Hashemi Nazari SS AU - Barzegar M AU - Ghazavi M AU - Beiraghi Toosi M AU - Nafissi S AU - Inaloo S AU - Zamani Ghaletaki G AU - Fatehi F AU - Heshmat R AU - Ghahvechi Akbari M AU - Abdi A AU - Bakhtiary H AU - Montazerlotfelahi H AU - Abbaskhanian A AU - Hosseini SA AU - Farshadmoghadam H AU - Hosseiny SMM AU - Shariatmadari F AU - Ziaadini B AU - Babaei M AU - Tavasoli A AU - Nikbakht S AU - Momen A AU - Khajeh A AU - Aminzadeh V AU - Mollamohammadi M AU - Taghdiri MM AU - Nasehi MM AU - Memarian S AU - Badv RS AU - Heidari M AU - Jafari N TI - Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data JO - Journal of neuromuscular diseases VL - 11 IS - 6 SP - 1190 EP - 1199 PY - 2024 ER -

Style

Citing Format

MLA

Ashrafi MR, et al.. "Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data." Journal of neuromuscular diseases, vol. 11, no. 6, 2024, pp. 1190-1199.

APA

Ashrafi MR, Babaee M, Hashemi Nazari SS, Barzegar M, Ghazavi M, Beiraghi Toosi M, Nafissi S, Inaloo S, Zamani Ghaletaki G, Fatehi F, Heshmat R, Ghahvechi Akbari M, Abdi A, Bakhtiary H, Montazerlotfelahi H, Abbaskhanian A, Hosseini SA, Farshadmoghadam H, Hosseiny SMM, ... Jafari N (2024). Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data. Journal of neuromuscular diseases, 11(6), 1190-1199.

Chicago

Ashrafi MR, Babaee M, Hashemi Nazari SS, Barzegar M, Ghazavi M, Beiraghi Toosi M, Nafissi S, et al.. "Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data." Journal of neuromuscular diseases 11, no. 6 (2024): 1190-1199.

Harvard

Ashrafi MR et al. (2024) 'Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data', Journal of neuromuscular diseases, 11(6), pp. 1190-1199.

Vancouver

Ashrafi MR, Babaee M, Hashemi Nazari SS, Barzegar M, Ghazavi M, Beiraghi Toosi M, et al.. Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data. Journal of neuromuscular diseases. 2024;11(6):1190-1199.

BibTex

@article{ author = {Ashrafi MR and Babaee M and Hashemi Nazari SS and Barzegar M and Ghazavi M and Beiraghi Toosi M and Nafissi S and Inaloo S and Zamani Ghaletaki G and Fatehi F and Heshmat R and Ghahvechi Akbari M and Abdi A and Bakhtiary H and Montazerlotfelahi H and Abbaskhanian A and Hosseini SA and Farshadmoghadam H and Hosseiny SMM and Shariatmadari F and Ziaadini B and Babaei M and Tavasoli A and Nikbakht S and Momen A and Khajeh A and Aminzadeh V and Mollamohammadi M and Taghdiri MM and Nasehi MM and Memarian S and Badv RS and Heidari M and Jafari N}, title = {Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data}, journal = {Journal of neuromuscular diseases}, volume = {11}, number = {6}, pages = {1190-1199}, year = {2024} }

RIS

TY - JOUR
AU - Ashrafi MR
AU - Babaee M
AU - Hashemi Nazari SS
AU - Barzegar M
AU - Ghazavi M
AU - Beiraghi Toosi M
AU - Nafissi S
AU - Inaloo S
AU - Zamani Ghaletaki G
AU - Fatehi F
AU - Heshmat R
AU - Ghahvechi Akbari M
AU - Abdi A
AU - Bakhtiary H
AU - Montazerlotfelahi H
AU - Abbaskhanian A
AU - Hosseini SA
AU - Farshadmoghadam H
AU - Hosseiny SMM
AU - Shariatmadari F
AU - Ziaadini B
AU - Babaei M
AU - Tavasoli A
AU - Nikbakht S
AU - Momen A
AU - Khajeh A
AU - Aminzadeh V
AU - Mollamohammadi M
AU - Taghdiri MM
AU - Nasehi MM
AU - Memarian S
AU - Badv RS
AU - Heidari M
AU - Jafari N
TI - Comparative Efficacy of Risdiplam and Nusinersen in Type 2 and 3 Spinal Muscular Atrophy Patients: A Cohort Study Using Real-World Data
JO - Journal of neuromuscular diseases
VL - 11
IS - 6
SP - 1190
EP - 1199
PY - 2024
ER -

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