Development of an International SMA Bulbar Assessment for Inter-professional Administration
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Development of an International SMA Bulbar Assessment for Inter-professional Administration. / Young, Sally Dunaway; McGrattan, Katlyn; Johnson, Emily; van der Heul, Marise; Duong, Tina; Bakke, Merete; Werlauff, Ulla; Pasternak, Amy; Cattaneo, Camilla; Hoffman, Katie; Fanelli, Lavinia; Breaks, Anne; Allison, Kristen; Baranello, Giovanni; Finkel, Richard; Coratti, Giorgia; Lofra, Robert Muni.
In: Journal of Neuromuscular Diseases, Vol. 10, No. 4, 2023, p. 639-652.Research output: Contribution to journal › Journal article › Research › peer-review
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TY - JOUR
T1 - Development of an International SMA Bulbar Assessment for Inter-professional Administration
AU - Young, Sally Dunaway
AU - McGrattan, Katlyn
AU - Johnson, Emily
AU - van der Heul, Marise
AU - Duong, Tina
AU - Bakke, Merete
AU - Werlauff, Ulla
AU - Pasternak, Amy
AU - Cattaneo, Camilla
AU - Hoffman, Katie
AU - Fanelli, Lavinia
AU - Breaks, Anne
AU - Allison, Kristen
AU - Baranello, Giovanni
AU - Finkel, Richard
AU - Coratti, Giorgia
AU - Lofra, Robert Muni
PY - 2023
Y1 - 2023
N2 - BACKGROUND: Progressive weakness can affect bulbar muscles in individuals with moderate to severe forms of spinal muscular atrophy (SMA). The paucity of standardized, valid bulbar assessments capturing clinically significant deficits in SMA impedes the ability to monitor function, facilitate intervention, or detect treatment response.OBJECTIVE: To fill this void, an international multidisciplinary team gathered to develop an agreed upon consensus-derived assessment of bulbar function in SMA for inter-professional administration to enhance our ability to monitor disease progression, support clinical management, and evaluate treatment effects.METHODS: Fifty-six international clinicians experienced in SMA were invited and engaged using the Delphi method over multiple rounds of web-based surveys to establish consensus.RESULTS: Serial virtual meetings occurred with 42 clinicians (21 speech and language therapists, 11 physical therapists, 5 neurologists, 4 occupational therapists, and 1 dentist). Seventy-two validated assessments of bulbar function were identified for potential relevance to individuals with SMA (32 accessible objective, 11 inaccessible objective, 29 patient-reported outcomes). Delphi survey rounds (n = 11, 15, 15) achieved consensus on individual items with relevance and wording discussed. Key aspects of bulbar function identified included: oral intake status, oral facial structure and motor strength, swallowing physiology, voice & speech, and fatigability.CONCLUSIONS: Multidisciplinary clinicians with expertise in bulbar function and SMA used Delphi methodology to reach consensus on assessments/items considered relevant for SMA across all age groups. Future steps include piloting the new scale moving towards validation/reliability. This work supports the advancement of assessing bulbar function in children and adults with SMA by a variety of professionals.
AB - BACKGROUND: Progressive weakness can affect bulbar muscles in individuals with moderate to severe forms of spinal muscular atrophy (SMA). The paucity of standardized, valid bulbar assessments capturing clinically significant deficits in SMA impedes the ability to monitor function, facilitate intervention, or detect treatment response.OBJECTIVE: To fill this void, an international multidisciplinary team gathered to develop an agreed upon consensus-derived assessment of bulbar function in SMA for inter-professional administration to enhance our ability to monitor disease progression, support clinical management, and evaluate treatment effects.METHODS: Fifty-six international clinicians experienced in SMA were invited and engaged using the Delphi method over multiple rounds of web-based surveys to establish consensus.RESULTS: Serial virtual meetings occurred with 42 clinicians (21 speech and language therapists, 11 physical therapists, 5 neurologists, 4 occupational therapists, and 1 dentist). Seventy-two validated assessments of bulbar function were identified for potential relevance to individuals with SMA (32 accessible objective, 11 inaccessible objective, 29 patient-reported outcomes). Delphi survey rounds (n = 11, 15, 15) achieved consensus on individual items with relevance and wording discussed. Key aspects of bulbar function identified included: oral intake status, oral facial structure and motor strength, swallowing physiology, voice & speech, and fatigability.CONCLUSIONS: Multidisciplinary clinicians with expertise in bulbar function and SMA used Delphi methodology to reach consensus on assessments/items considered relevant for SMA across all age groups. Future steps include piloting the new scale moving towards validation/reliability. This work supports the advancement of assessing bulbar function in children and adults with SMA by a variety of professionals.
U2 - 10.3233/JND-221672
DO - 10.3233/JND-221672
M3 - Journal article
C2 - 37212069
VL - 10
SP - 639
EP - 652
JO - Journal of Neuromuscular Diseases
JF - Journal of Neuromuscular Diseases
SN - 0960-8966
IS - 4
ER -
ID: 347739484