“I have SMA, SMA doesn’t have me”- A Qualitative Snapshot into the Challenges, Successes, and Quality of Life of Adolescents and Young Adults with SMA
Background: With the approval of three treatments for spinal muscular atrophy (SMA) and several promising therapies on the horizon, the SMA adolescent and young adult populations are expected to evolve in the coming years. It is imperative to understand this cohort as it exists today to provide optimal care and resources, as well as to assess possible treatment effects over time. In 2018, Cure SMA launched two initiatives geared towards understanding adolescents and young adults with SMA, ages 12-25. First, Cure SMA launched a Quality of Life (QoL) survey to capture quantitative and qualitative information on this specific age demographic. Concurrently, Cure SMA invited SMA-affected individuals, ages 12-25, to create a three-minute video on their everyday experiences living with SMA. An inductive thematic analysis of the free-text survey questions along with the video contest findings are reported here.
Results: Eighty-five individuals — 6 type Is, 58 type IIs, and 21 type IIIs — completed the Quality of Life free-response, while six individuals participated in the SMA awareness video contest. In both settings, individuals detailed a variety of challenges, including but not limited to forming or maintaining close relationships, experiencing feelings of isolation, challenges with accessibility, independence, and dealing with the stigma of being perceived as mentally disabled. Individuals also discussed their successes, including but not limited to higher education enrollment and attendance, development of quality friendships, and perseverance through obstacles. Additionally, notably in the survey, 39% of respondents requested the creation of an SMA peer support group in efforts to connect with each other as well as collectively navigate the aforementioned challenges they face.
Conclusion: Together, these findings provide a rare glimpse into the unique mindsets, challenges and motivations of SMA adolescents and young adults, via patient-reported measures instead of caregiver proxy. The adolescent and young adult age demographics assessed represent a critical transition period in life and in SMA care. No one understands the needs of an adolescent or young adult with SMA better than the individuals themselves, and it is critical to encapsulate their insights to affect change.
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Posted 16 Dec, 2020
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“I have SMA, SMA doesn’t have me”- A Qualitative Snapshot into the Challenges, Successes, and Quality of Life of Adolescents and Young Adults with SMA
Posted 16 Dec, 2020
On 11 Dec, 2020
Received 10 Dec, 2020
Received 08 Dec, 2020
On 01 Dec, 2020
On 30 Nov, 2020
Invitations sent on 30 Nov, 2020
On 30 Nov, 2020
On 30 Nov, 2020
On 30 Nov, 2020
On 03 Nov, 2020
Received 02 Nov, 2020
Received 29 Oct, 2020
On 18 Oct, 2020
Invitations sent on 15 Oct, 2020
On 15 Oct, 2020
On 14 Oct, 2020
On 13 Oct, 2020
On 13 Oct, 2020
On 12 Sep, 2020
Received 11 Sep, 2020
On 26 Aug, 2020
Received 25 Aug, 2020
Invitations sent on 10 Aug, 2020
On 10 Aug, 2020
On 28 Jul, 2020
On 27 Jul, 2020
On 27 Jul, 2020
On 27 Jul, 2020
Background: With the approval of three treatments for spinal muscular atrophy (SMA) and several promising therapies on the horizon, the SMA adolescent and young adult populations are expected to evolve in the coming years. It is imperative to understand this cohort as it exists today to provide optimal care and resources, as well as to assess possible treatment effects over time. In 2018, Cure SMA launched two initiatives geared towards understanding adolescents and young adults with SMA, ages 12-25. First, Cure SMA launched a Quality of Life (QoL) survey to capture quantitative and qualitative information on this specific age demographic. Concurrently, Cure SMA invited SMA-affected individuals, ages 12-25, to create a three-minute video on their everyday experiences living with SMA. An inductive thematic analysis of the free-text survey questions along with the video contest findings are reported here.
Results: Eighty-five individuals — 6 type Is, 58 type IIs, and 21 type IIIs — completed the Quality of Life free-response, while six individuals participated in the SMA awareness video contest. In both settings, individuals detailed a variety of challenges, including but not limited to forming or maintaining close relationships, experiencing feelings of isolation, challenges with accessibility, independence, and dealing with the stigma of being perceived as mentally disabled. Individuals also discussed their successes, including but not limited to higher education enrollment and attendance, development of quality friendships, and perseverance through obstacles. Additionally, notably in the survey, 39% of respondents requested the creation of an SMA peer support group in efforts to connect with each other as well as collectively navigate the aforementioned challenges they face.
Conclusion: Together, these findings provide a rare glimpse into the unique mindsets, challenges and motivations of SMA adolescents and young adults, via patient-reported measures instead of caregiver proxy. The adolescent and young adult age demographics assessed represent a critical transition period in life and in SMA care. No one understands the needs of an adolescent or young adult with SMA better than the individuals themselves, and it is critical to encapsulate their insights to affect change.
Figure 1
Figure 2
Figure 3
Figure 4
Figure 5