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Pregled bibliografske jedinice broj: 1259102

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Mercuri, Eugenio; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Boespflug-Tanguy, Odile; Goemans, Nathalie et al.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial // The Lancet Neurology, 21 (2022), 1; 42-52 doi:10.1016/s1474-4422(21)00367-7 (međunarodna recenzija, članak, znanstveni)

CROSBI ID: 1259102 Za ispravke kontaktirajte CROSBI podršku putem web obrasca

Naslov
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Autori
Mercuri, Eugenio ; Deconinck, Nicolas ; Mazzone, Elena S ; Nascimento, Andres ; Oskoui, Maryam ; Saito, Kayoko ; Vuillerot, Carole ; Baranello, Giovanni ; Boespflug-Tanguy, Odile ; Goemans, Nathalie ; Kirschner, Janbernd ; Kostera- Pruszczyk, Anna ; Servais, Laurent ; Gerber, Marianne ; Gorni, Ksenija ; Khwaja, Omar ; Kletzl, Heidemarie ; Scalco, Renata S ; Staunton, Hannah ; Yeung, Wai Yin ; Martin, Carmen ; Fontoura, Paulo ; Day, John W ; Volpe, Joseph J. ; Posner, John ; Kellner, Ulrich ; Quinlivan, Rosaline ; Daron, Aurore ; Delstanche, Stéphanie ; Bruninx, Romain ; Dal Farra, Fabian ; Schneider, Olivier ; Deconinck, Nicolas ; Balikova, Irina ; Delbeke, Patricia ; Joniau, Inge ; Tahon, Valentine ; Wittevrongel, Sylvia ; De Vos, Elke ; Goemans, Nathalie ; Casteels, Ingele ; De Waele, Liesbeth ; Balikova, Irina ; Cassiman, Catherine ; Prové, Lies ; Kinoo, David ; Vancampenhout, Lisa ; Van Den Hauwe, Marleen ; Van Impe, Annelies ; Prufer de Queiroz Campos Araujo, Alexandra ; Chacon Pereira, Aline ; Nardes, Flávia ; Haefeli, Lorena ; Rossetto, Julia ; Ferreira Rebel, Marcos ; Almeida Pereira, Jaqueline ; Campbell, Craig ; Sharan, Sapna ; McDonald, Wendy ; Scholtes, Cheryl ; Mah, Jean ; Sframeli, Maria ; Chiu, Angela ; Hagel, Jane ; Oskoui, Maryam ; Beneish, Raquel ; Cariou-Palmer, Gaela ; Pham, Connie ; Toffoli, Daniela ; Arpin, Stephanie ; Turgeon Desilets, Sarah ; Wang, Yi ; Hu, Chaoping ; Huan, Jianfeng ; Qian, Chen ; Shen, Li ; Xiao, Ying ; Zhou, Zhenxuan ; Li, Hui ; Wang, Sujuan ; Xiong, Hui ; Chang, Xingzhi ; Dong, Hui ; Liu, Ying ; Sang, Tian ; Wei, Cuijie ; Wen, Jing ; Cao, Yiwen ; Ly, Xingyao ; Wen, Jing ; Zhao, Jingjing ; Li, Wenzhu ; Qin, Lun ; Barisic, Nina ; Galiot Delic, Martina ; Ivkic, Petra Kristina ; Vukojevic, Nenad ; Kern, Ivana ; Najdanovic, Boris ; Skugor, Marin ; Servais, Laurent ; Boespflug-Tanguy, Odile ; Gidaro, Teresa ; Seferian, Andreea ; De Lucia, Silvana ; Barreau, Emmanuel ; Mnafek, Nabila ; Momtchilova, Marta Milkova ; Peche, Helene ; Valherie, Carole ; Grange, Allison ; Lilien, Charlotte ; Milascevic, Darko ; Tachibana, Shotaro ; Ravelli, Claudia ; Cardas, Ruxandra ; Taytard, Jessica ; Aubertin, Guillaume ;

Izvornik
The Lancet Neurology (1474-4422) 21 (2022), 1; 42-52

Vrsta, podvrsta i kategorija rada
Radovi u časopisima, članak, znanstveni

Ključne riječi
Risdiplam ; spinal muscular atrophy

Sažetak
Findings: Between Oct 9, 2017, and Sept 4, 2018, 180 patients were randomly assigned to receive risdiplam (n=120) or placebo (n=60). For analysis of the primary endpoint, 115 patients from the risdiplam group and 59 patients from the placebo group were included. At month 12, the least squares mean change from baseline in 32-item Motor Function Measure was 1·36 (95% CI 0·61 to 2·11) in the risdiplam group and -0·19 (-1·22 to 0·84) in the placebo group, with a treatment difference of 1·55 (0·30 to 2·81, p=0·016) in favour of risdiplam. 120 patients who received risdiplam and 60 who received placebo were included in safety analyses. Adverse events that were reported in at least 5% more patients who received risdiplam than those who received placebo were pyrexia (25 [21%] of 120 patients who received risdiplam vs ten [17%] of 60 patients who received placebo), diarrhoea (20 [17%] vs five [8%]), rash (20 [17%] vs one [2%]), mouth and aphthous ulcers (eight [7%] vs 0), urinary tract infection (eight [7%] vs 0), and arthralgias (six [5%] vs 0). The incidence of serious adverse events was similar between treatment groups (24 [20%] of 120 patients in the risdiplam group ; 11 [18%] of 60 patients in the placebo group), with the exception of pneumonia (nine [8%] in the risdiplam group ; one [2%] in the placebo group). Interpretation: Risdiplam resulted in a significant improvement in motor function compared with placebo in patients aged 2- 25 years with type 2 or non-ambulant type 3 spinal muscular atrophy. Our exploratory subgroup analyses showed that motor function was generally improved in younger individuals and stabilised in older individuals, which requires confirmation in further studies. SUNFISH part 2 is ongoing and will provide additional evidence regarding the long-term safety and efficacy of risdiplam.

Izvorni jezik
Engleski



POVEZANOST RADA

Ustanove:
Medicinski fakultet, Zagreb

Profili:

Avatar Url Nenad Vukojević (autor)

Avatar Url Nina Barišić (autor)

Poveznice na cjeloviti tekst rada:

doi

Citiraj ovu publikaciju:

Mercuri, Eugenio; Deconinck, Nicolas; Mazzone, Elena S; Nascimento, Andres; Oskoui, Maryam; Saito, Kayoko; Vuillerot, Carole; Baranello, Giovanni; Boespflug-Tanguy, Odile; Goemans, Nathalie et al.
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial // The Lancet Neurology, 21 (2022), 1; 42-52 doi:10.1016/s1474-4422(21)00367-7 (međunarodna recenzija, članak, znanstveni)
Mercuri, E., Deconinck, N., Mazzone, E., Nascimento, A., Oskoui, M., Saito, K., Vuillerot, C., Baranello, G., Boespflug-Tanguy, O. & Goemans, N. (2022) Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial. The Lancet Neurology, 21 (1), 42-52 doi:10.1016/s1474-4422(21)00367-7.
@article{article, author = {Mercuri, Eugenio and Deconinck, Nicolas and Mazzone, Elena S and Nascimento, Andres and Oskoui, Maryam and Saito, Kayoko and Vuillerot, Carole and Baranello, Giovanni and Boespflug-Tanguy, Odile and Goemans, Nathalie and Kirschner, Janbernd and Kostera- Pruszczyk, Anna and Servais, Laurent and Gerber, Marianne and Gorni, Ksenija and Khwaja, Omar and Kletzl, Heidemarie and Scalco, Renata S and Staunton, Hannah and Yeung, Wai Yin and Martin, Carmen and Fontoura, Paulo and Day, John W and Volpe, Joseph J. and Posner, John and Kellner, Ulrich and Quinlivan, Rosaline and Daron, Aurore and Delstanche, St\'{e}phanie and Bruninx, Romain and Dal Farra, Fabian and Schneider, Olivier and Deconinck, Nicolas and Balikova, Irina and Delbeke, Patricia and Joniau, Inge and Tahon, Valentine and Wittevrongel, Sylvia and De Vos, Elke and Goemans, Nathalie and Casteels, Ingele and De Waele, Liesbeth and Balikova, Irina and Cassiman, Catherine and Prov\'{e}, Lies and Kinoo, David and Vancampenhout, Lisa and Van Den Hauwe, Marleen and Van Impe, Annelies and Prufer de Queiroz Campos Araujo, Alexandra and Chacon Pereira, Aline and Nardes, Fl\'{a}via and Haefeli, Lorena and Rossetto, Julia and Ferreira Rebel, Marcos and Almeida Pereira, Jaqueline and Campbell, Craig and Sharan, Sapna and McDonald, Wendy and Scholtes, Cheryl and Mah, Jean and Sframeli, Maria and Chiu, Angela and Hagel, Jane and Oskoui, Maryam and Beneish, Raquel and Cariou-Palmer, Gaela and Pham, Connie and Toffoli, Daniela and Arpin, Stephanie and Turgeon Desilets, Sarah and Wang, Yi and Hu, Chaoping and Huan, Jianfeng and Qian, Chen and Shen, Li and Xiao, Ying and Zhou, Zhenxuan and Li, Hui and Wang, Sujuan and Xiong, Hui and Chang, Xingzhi and Dong, Hui and Liu, Ying and Sang, Tian and Wei, Cuijie and Wen, Jing and Cao, Yiwen and Ly, Xingyao and Wen, Jing and Zhao, Jingjing and Li, Wenzhu and Qin, Lun and Barisic, Nina and Galiot Delic, Martina and Ivkic, Petra Kristina and Vukojevic, Nenad and Kern, Ivana and Najdanovic, Boris and Skugor, Marin and Servais, Laurent and Boespflug-Tanguy, Odile and Gidaro, Teresa and Seferian, Andreea and De Lucia, Silvana and Barreau, Emmanuel and Mnafek, Nabila and Momtchilova, Marta Milkova and Peche, Helene and Valherie, Carole and Grange, Allison and Lilien, Charlotte and Milascevic, Darko and Tachibana, Shotaro and Ravelli, Claudia and Cardas, Ruxandra and Taytard, Jessica and Aubertin, Guillaume}, year = {2022}, pages = {42-52}, DOI = {10.1016/s1474-4422(21)00367-7}, keywords = {Risdiplam, spinal muscular atrophy}, journal = {The Lancet Neurology}, doi = {10.1016/s1474-4422(21)00367-7}, volume = {21}, number = {1}, issn = {1474-4422}, title = {Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial}, keyword = {Risdiplam, spinal muscular atrophy} }
@article{article, author = {Mercuri, Eugenio and Deconinck, Nicolas and Mazzone, Elena S and Nascimento, Andres and Oskoui, Maryam and Saito, Kayoko and Vuillerot, Carole and Baranello, Giovanni and Boespflug-Tanguy, Odile and Goemans, Nathalie and Kirschner, Janbernd and Kostera- Pruszczyk, Anna and Servais, Laurent and Gerber, Marianne and Gorni, Ksenija and Khwaja, Omar and Kletzl, Heidemarie and Scalco, Renata S and Staunton, Hannah and Yeung, Wai Yin and Martin, Carmen and Fontoura, Paulo and Day, John W and Volpe, Joseph J. and Posner, John and Kellner, Ulrich and Quinlivan, Rosaline and Daron, Aurore and Delstanche, St\'{e}phanie and Bruninx, Romain and Dal Farra, Fabian and Schneider, Olivier and Deconinck, Nicolas and Balikova, Irina and Delbeke, Patricia and Joniau, Inge and Tahon, Valentine and Wittevrongel, Sylvia and De Vos, Elke and Goemans, Nathalie and Casteels, Ingele and De Waele, Liesbeth and Balikova, Irina and Cassiman, Catherine and Prov\'{e}, Lies and Kinoo, David and Vancampenhout, Lisa and Van Den Hauwe, Marleen and Van Impe, Annelies and Prufer de Queiroz Campos Araujo, Alexandra and Chacon Pereira, Aline and Nardes, Fl\'{a}via and Haefeli, Lorena and Rossetto, Julia and Ferreira Rebel, Marcos and Almeida Pereira, Jaqueline and Campbell, Craig and Sharan, Sapna and McDonald, Wendy and Scholtes, Cheryl and Mah, Jean and Sframeli, Maria and Chiu, Angela and Hagel, Jane and Oskoui, Maryam and Beneish, Raquel and Cariou-Palmer, Gaela and Pham, Connie and Toffoli, Daniela and Arpin, Stephanie and Turgeon Desilets, Sarah and Wang, Yi and Hu, Chaoping and Huan, Jianfeng and Qian, Chen and Shen, Li and Xiao, Ying and Zhou, Zhenxuan and Li, Hui and Wang, Sujuan and Xiong, Hui and Chang, Xingzhi and Dong, Hui and Liu, Ying and Sang, Tian and Wei, Cuijie and Wen, Jing and Cao, Yiwen and Ly, Xingyao and Wen, Jing and Zhao, Jingjing and Li, Wenzhu and Qin, Lun and Barisic, Nina and Galiot Delic, Martina and Ivkic, Petra Kristina and Vukojevic, Nenad and Kern, Ivana and Najdanovic, Boris and Skugor, Marin and Servais, Laurent and Boespflug-Tanguy, Odile and Gidaro, Teresa and Seferian, Andreea and De Lucia, Silvana and Barreau, Emmanuel and Mnafek, Nabila and Momtchilova, Marta Milkova and Peche, Helene and Valherie, Carole and Grange, Allison and Lilien, Charlotte and Milascevic, Darko and Tachibana, Shotaro and Ravelli, Claudia and Cardas, Ruxandra and Taytard, Jessica and Aubertin, Guillaume}, year = {2022}, pages = {42-52}, DOI = {10.1016/s1474-4422(21)00367-7}, keywords = {Risdiplam, spinal muscular atrophy}, journal = {The Lancet Neurology}, doi = {10.1016/s1474-4422(21)00367-7}, volume = {21}, number = {1}, issn = {1474-4422}, title = {Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial}, keyword = {Risdiplam, spinal muscular atrophy} }

Časopis indeksira:


  • Current Contents Connect (CCC)
  • Web of Science Core Collection (WoSCC)
    • Science Citation Index Expanded (SCI-EXP)
    • SCI-EXP, SSCI i/ili A&HCI
  • Scopus
  • MEDLINE

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