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Center for Health + Technology [CHeT]

URMC / Center for Health + Technology / Our Expertise / CHeT Outcomes / SMA-HI: The Spinal Muscular Atrophy Health Index

SMA-HI: The Spinal Muscular Atrophy Health Index

  • Type of Clinical Outcome Assessment (COA): Patient-reported outcome measure (PRO) and Caregiver or observer-reported outcome (CR/ObsRO)
  • Administration Mode: Self-administered
  • Therapeutic Area: Spinal Muscular Atrophy
  • Approximate Completion Time: <15 minutes
  • Required Supervision: None
  • Recall Period: Immediate point in time

Instrument Variations

  • The Spinal Muscular Atrophy Health Index Short Form (SMA-HI-SF)
    • SMA-HI Short Form Completion Time: Under 1 minute
  • The Spinal Muscular Atrophy Caregiver Reported Health Index (SMACR-HI) 0-18
    • SMACR-HI 0-18 Completion Time: <5 minutes (completed by a caregiver on behalf of a patient)
  • The Spinal Muscular Atrophy Caregiver Reported Health Index Short Form (SMACR-HI-SF) 0-18
    • SMACR-HI 0-18 Short Form Completion Time: Under 1 minute (completed by a caregiver on behalf of a patient)
  • The Spinal Muscular Atrophy Caregiver Reported Health Index (SMACR-HI) 5-18
    • SMACR-HI 5-18 Completion Time: <10 minutes (completed by a caregiver on behalf of a patient)
  • The Spinal Muscular Atrophy Caregiver Reported Health Index Short Form (SMACR-HI-SF) 5-18
    • SMACR-HI 5-18 Short Form Completion Time: Under 1 minute (completed by a caregiver on behalf of a patient)

Symptom Subscales

  • SMA-HI Number of Independently Validated Symptom Subscales: 14
    • SMA-HI Symptom Subscales: Hip, Thigh & Knee Function; Shoulder & Arm Function; Back, Chest & Abdominal Function; Social Performance; Fatigue; Activity Participation; Hand & Finger Strength; Social Satisfaction; Emotional Health; Pain; Breathing Function; Swallowing Function; Sleep; Gastrointestinal Function; Mobility & Ambulation (Supplemental Scale)
  • SMACR-HI 0-18 Number of Independently Validated Symptom Subscales: 8
    • SMACR-HI 0-18 Symptom Subscales: Hip, Thigh & Knee Function; Activity Participation; Back, Chest & Abdominal Function; Shoulder & Arm Function; Swallowing Function; Hand & Finger Strength; Breathing Function; Sleep & Daytime Sleepiness
  • SMACR-HI 5-18 Number of Independently Validated Symptom Subscales: 13
    • SMACR-HI 5-18 Symptom Subscales: Back, Chest & Abdominal Function; Shoulder & Arm Function; Hand & Finger Strength; Breathing Function; Activity Participation; Emotional Health; Fatigue; Gastrointestinal Function; Pain; Sleep & Daytime Sleepiness; Communication; Hip, Thigh & Knee Function; Swallowing Function; Mobility & Ambulation (Supplemental Scale)

Instrument Attributes

  • The SMA-HI is a patient-reported outcome (PRO) measure designed to assess symptoms and health-related quality-of-life from the perspective of patients with spinal muscular atrophy.
  • Designed and validated to fully satisfy regulatory and published FDA guidance for use in drug-labeling claims and measuring changes in how a patient feels and functions.
  • In a multicenter clinical study examining Spinraza efficacy in adults with SMA, the SMA-HI showed an improvement in total disease burden over 14 months that was undetected by traditional measures. [Zaidman et al. (2023). Spinraza in Adults with Spinal Muscular Atrophy (SAS) – 14 month results (S33.010). Neurology. 100. 3622. 10.1212/WNL.0000000000203405.]
  • The SMA-HI is a fully valid, reliable, responsive, and disease-specific instrument capable of measuring changes in patient-reported health in patients with spinal muscular atrophy in response to therapeutic intervention during clinical trials or clinical monitoring.
  • The SMA-HI was developed using extensive patient input, including an analysis of 1,045 patient quotes and a large cross-sectional study involving 359 patients.
  • The SMA-HI is highly relevant to patients, has low patient burden, correlates with markers of disease severity, and demonstrates no floor or ceiling effects.
  • The SMACR-HI 0-18 and SMACR-HI 5-18 are caregiver-reported outcome (CRO/ObsRO) measures designed to assess symptoms and health-related quality-of-life from the perspective of caregivers of individuals with spinal muscular atrophy ages 0-18 and 5-18. The SMACR-HI 0-18 demonstrates few floor and no ceiling effects, and the SMACR-HI 5-18 demonstrates no floor or ceiling effects.
  • The SMACR-HI was developed using extensive caregiver input, including an analysis of 670 caregiver quotes and a large cross-sectional study involving 77 patients.
  • The SMACR-HI is highly relevant to patients, has low patient burden, and correlates with markers of disease severity.
  • Used in industry, academic, and foundation studies.
  • English
  • French (CA)
  • Italian

Additional translations available upon request.

  • Hunter, M; Heatwole, C; Luebbe, E; Johnson, N.  What Matters Most: A Perspective From Adult Spinal Muscular Atrophy Patients.  Journal of Neuromuscular Diseases.  2016 Aug 30;3(3):425-429.
  • Guber, RD; Kokkinis, AD; Schindler, AB; Bendixen, RM; Heatwole, CR; Fischbeck, KH; Grunseich, C.  Patient-Identified Impact of Symptoms in Spinal and Bulbar Muscular Atrophy.  Muscle Nerve. 2018 Jan;57(1):40-44.
  • Mongiovi P; Dilek N; Garland C; Hunter M; Kissel J; Luebbe E; McDermott M; Johnson N; Heatwole C.  Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA).  Neurology. 2018;91(13):e1206-e1214.
  • Zizzi CE, Luebbe E, Mongiovi P, Hunter M, Dilek N, Garland C, Ciafaloni E, Zaidman CM, Kissel JT, McDermott MP, Johnson N, Sansone V, Heatwole CR. The Spinal Muscular Atrophy Health Index: A novel outcome for measuring how a patient feels and functions. Muscle Nerve. 2021 Jun;63(6):837-844. doi: 10.1002/mus.27223. Epub 2021 Mar 24. PMID: 33711174.
  • Sansone V, Pirola A, Lizio A, Greco L, Coratti G, Casiraghi J, Pane M, Pera M, Italiano C, Messina S, Pozzi S, Sframeli M, D’Amico A, Bertini E, Claudio B, Mauro L, Samin F, Albamonte E, Zizzi C, Heatwole C, Eugenio M.  The Spinal Muscular Atrophy Health Index:  Italian Validation of a Disease-Specific Outcome Measure.  Neuromuscular Disorders.  2021.
  • Mazzella A, Cruz R, Belter L, Curry M, Dilek N, Zizzi C, Heatwole C, Jarecki J. Assessing perspectives of disease burden and clinically meaningful changes using the Spinal Muscular Atrophy Health Index in adolescents and young adults. Muscle Nerve. 2022;66(3):276-281. doi:10.1002/mus.27644.
  • Zaidman, C., Proud, C., Thonhoff, J., Rad, N., Ho, D., Chu, M., Ladha, S., Crawford, T., Nayar, S., Genge, A., Frey, M., Heatwole, C., & Lew, D. (2023). Spinraza in adults with spinal muscular atrophy (SAS) – 14 month results (S33.010). Neurology, 100(17_supplement_2). https://doi.org/10.1212/wnl.0000000000203405.
  • Belter L, Peterson I, Jarecki J. Evaluating Perceived Fatigue within an Adult Spinal Muscular Atrophy Population. Neurol Ther. 2023 Dec;12(6):2161–2175. doi:10.1007/s40120-023-00552-y. PMCID: PMC10630176. PMID: 37856000. Epub 2023 Oct 19.
  • “Measuring Clinically Relevant Change in Adult Spinal Muscular Atrophy Clinical Trials: The development of the SMA Health Index.”  Kansas City, Kansas.  Cure SMA Research Meeting. 6/17/15.
  • “What Matters Most: A Perspective From Adult Spinal Muscular Atrophy Patients.” Kansas City, Kansas.  Cure SMA Family and Researchers Session. 6/17/15.
  • “Patient-Identified Disease Burden in Adult Spinal Muscular Atrophy.”  Kansas City, Kansas.  Cure SMA Research Meeting. 6/19/15.
  • “Adults Living With SMA: A Patient-Centered Assessment of the Symptoms that Matter Most.”  Chad Heatwole, Michael Hunter, Liz Luebbe, Connie Garland, Nicholas Johnson.  Cure SMA Foundation.  Anaheim, CA.  6/2016.
  •  “PRISM-SMA: Patient Reported Impact of Symptoms in Adult Spinal Muscular Atrophy.”  Anaheim, CA.  Cure SMA Research Meeting.  Clinical Science Session.  6/16/16.
  •  “PRISM-SMA: Patient reported impact of symptoms in adult Spinal Muscular Atrophy.”  Muscle Study Group Annual Meeting.  Salt Lake City, UT.  9/25/16.
  • “Development of a Disease-Specific Patient-Reported Outcome Measure for Spinal Muscular Atrophy Patients: The Spinal Muscular Atrophy Health Index (SMA-HI).”  Chad Heatwole, Michael Hunter, Connie Garland, Nicholas Johnson, Philip Mongiovi, Elizabeth Luebbe.  CURESMA Meeting.  Orlando, FL.  7/2017.
  • “Measuring Therapeutic Benefit in Clinical Trials: The SMA-Health Index.”  Mongiovi, P.  Dilek, N.  Garland, C.  Hunger, M.  Luebbe, E.  McDermott, M.  Johnson, N.  Kissel, J.  Heatwole, C.  CureSMA patient/researcher session.  Dallas, TX.  6/2018.
  •  “Assessing Clinical Meaningfulness and Patient Experience in the SMA Teen and Young Adult Populations.” A. Mazzella, M. Curry, R. Cruz, C. Heatwole, C. Zizzi, L. Belter, J. Jarecki. June 2019. 2019 Cure SMA Conference.
  •  “The Spinal Muscular Atrophy Caregiver Reported Health Index (SMACR-HI): An Valid Observer Reported Outcome Measure.”  CureSMA industry meeting.  Anaheim, CA.  6/27/19.
  • “The Spinal Muscular Atrophy Caregiver Reported Health Index (SMACR-HI): An Valid Observer Reported Outcome Measure.”  SMA Industry Conference Hope on the Hill.  Washington DC.  11/8/19.
  • “The Spinal Muscular Atrophy Caregiver Reported Health Index (SMACR-HI): A Valid Observer Reported Outcome Measure.” Cure SMA Industry Collaboration Regulatory Topic Group Meeting. Virtual. 12/16/20.
  • “The Spinal Muscular Atrophy Caregiver Reported Health Index (SMACR-HI): A Valid Observer Reported Outcome Measure.” Cure SMA Industry Collaboration Regulatory Topic Group Meeting. Virtual. 2/17/21.
  • “The Spinal Muscular Atrophy Caregiver-Health Indices (SMACR-HI): Valid Outcomes for Measuring Changes in Disease Burden During Pediatric SMA Clinical Trials.” J. Seabury,  J. Weinstein, S. Rosero, C. Zizzi,  N. Dilek,  E. Luebbe, C. Heatwole. Cure SMA Virtual Research and Clinical Care Meeting. Virtual. 6/9/21.
  •  “The Spinal Muscular Atrophy Caregiver Reported Health Indices (SMACR-HI): Valid Observer Reported Outcome Measures”. Cure SMA Industry Collaboration Meeting. Virtual. 6/15/21. “The Spinal Muscular Atrophy Caregiver Reported Health Indices (SMACR-HI): Valid Observer Reported Outcome Measures.” Cure SMA Industry Collaboration Regulatory Topic Group Meeting. Virtual. 6/15/21.
  • “The Spinal Muscular Atrophy Caregiver Reported Health Indices (SMACR-HI): Valid Observer Reported Outcome Measures.” Cure SMA Industry Collaboration Regulatory Topic Group Meeting. Virtual. 8/18/21.
  • “The Spinal Muscular Atrophy Caregiver Reported Health Indices (SMACR-HI): Valid Observer Reported Outcome Measures.” Cure SMA Industry Collaboration Regulatory Topic Group Meeting. Virtual. 10/20/21.
  • “The Spinal Muscular Atrophy Caregiver Reported Health Indices (SMACR-HI): Disease-Specific Observer Reported Outcome Measures.” Cure SMA Industry Collaboration Regulatory Topic Group Meeting. Washington, DC. 12/1/21.
  • “The Spinal Muscular Atrophy Caregiver Reported Health Indices (SMACR-HI): Disease-Specific Observer Reported Outcome Measures.” Cure SMA Industry Collaboration Regulatory Topic Group Meeting. Virtual. 4/20/22.
  • “The Spinal Muscular Atrophy Caregiver-Health Indices: Development and Validation of Two Novel Caregiver Reported Outcome Measures for Clinical Trials.” S. Rosero, J. Seabury, A. Varma, C. Zizzi, J. Heatwole, E. Luebbe, P. Mongiovi, M. Hunter, N. Dilek, E.Ciafaloni, C. Zaidman, M. McDermott, N. Johnson, V. Sansone, C. Heatwole, SMA Research and Clinical Care Meeting, Anaheim, CA. 06/14/22.
  • “The Spinal Muscular Atrophy Caregiver-Health Indices: Development and Validation of Two Novel Caregiver Reported Outcome Measures for Clinical Trials.” S. Rosero, J. Seabury, J. Weinstein, A. Varma, C. Zizzi, J. Heatwole, E. Luebbe, P. Mongiovi, M. Hunter, N. Dilek, E. Ciafaloni, C. Zaidman, M. McDermott, N. Johnson, V. Sansone, C. Heatwole. Cure SMA Research and Clinical Care Meeting. Anaheim, CA. 6/16/22.
  • “Spinraza in Adults with Spinal Muscular Atrophy (SAS)- 14 month results.” C. Zaidman, C. Proud, J. Thonhoff, N. Rad, D. Ho, M. Chu, S. Ladha, T. Crawford, S. Nayar, A. Genge, M. Frey, C. Heatwole, D. Lew. American Academy of Neurology (AAN) Annual Meeting. Boston, MA. 4/22/23.
  • “The Spinal Muscular Atrophy-Health Index Short Form: Validation of a Novel Outcome Measure for Therapeutic Trials”. S. Rosero, A. Varma, C. Zizzi, E. Luebbe, J. Seabury, J. Weinstein, C. Engebrecht, S. Khosa, P. Mongiovi, M. Hunter, N. Dilek, C. Garland, E. Ciafaloni, C. Zaidman, J. Kissel, M. McDermott, N. Johnson, V. Sansone, C. Heatwole. Oral session presented at: 2023 Annual CureSMA Conference; June 29-July 2, 2023; Orlando FL.
  • “The Spinal Muscular Atrophy Caregiver-Health Indices Short Forms: Validation of Two Novel Outcome Measures for Use in Clinical Trials”.  S. Rosero, J. Weinstein, J. Seabury, A. Varma, C. Engebrecht, C. Zizzi, J. Heatwole, S. Khosa, D. Alexandrou, N. Dilek, B. Johnson, C. Heatwole. Poster session presented at: 2023 Annual SMA Conference; June 29-July2, 2023; Orlando, FL.

Instrument Scoring

All subscales are scored on a scale of 0 to 100 with 0 representing no disease burden and 100 representing the maximum level of disease burden. Symptom questions within each subscale are weighted based on participant-reported prevalence and average impact as identified through the cross-sectional study. Subscale scores are also weighted to generate a total SMA-HI or SMACR-HI score (0-100) representing overall disease burden.

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