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Center for Health + Technology [CHeT]

URMC / Center for Health + Technology / Our Expertise / CHeT Outcomes / MD2HI: The Myotonic Dystrophy Type 2 Health Index

MD2HI: The Myotonic Dystrophy Type 2 Health Index

  • Type of Clinical Outcome Assessment (COA): Patient-reported outcome measure (PRO)
  • Administration Mode: Self-administered
  • Therapeutic Area: Myotonic dystrophy type 2
  • Approximate Completion Time: 15 minutes
  • Required Supervision: None
  • Recall Period: Immediate point in time

Instrument Variations

  • The Myotonic Dystrophy Type 2 Health Index Short Form (MD2HI-SF)
    • MD2 Short From Completion Time: Under 1 minute

Symptom Subscales

  • Number of Independently Validated Symptom Subscales: 17

Mobility & Ambulation; Hip, Thigh & Knee Strength; Back, Chest & Abdominal Strength; Shoulder & Arm Function; Hand & Finger Function; Emotional Issues; Cognitive Function; Sleep & Daytime Sleepiness; Fatigue; Pain; Myotonia; Gastrointestinal Health; Swallowing Function; Activity Participation; Communication;  Social Satisfaction; Breathing Function

Instrument Attributes

  • The MD2HI is a patient-reported outcome (PRO) measure designed to assess symptoms and health-related quality-of-life from the perspective of patients with myotonic dystrophy type 2.
  • 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.
  • The MD2HI is a fully valid, reliable, responsive, and disease-specific instrument capable of measuring changes in patient-reported health in patients with myotonic dystrophy type 2.
  • The instrument was developed using extensive patient input, including an analysis of 943 patient quotes and a large cross-sectional study involving 74 patients.
  • The MD2HI is highly relevant to patients, has low patient burden, correlates with markers of disease severity, and demonstrates low floor and no ceiling effects.
  • Used in academic and industry sponsored clinical studies.
  • The MD2HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.97 for the full instrument).
  • English

Additional translations available upon request.

  • Heatwole, C; Johnson, N; Bode, R; Dekdebrun, J; Dilek, N; Hilbert, J; Luebbe, E; Martens, W; McDermott, M; Quinn, C; Rothrock, N; Thornton, C; Vickrey, B; Victorson, D; Moxley, R.  PRISM-2: Patient Reported Impact of Symptoms in Myotonic Dystrophy Type 2.  Neurology.  2015 Dec 15;85(24):2136-46
  • “Myotonic Dystrophy Type-2: Patient-Reported Highest-Impact Symptoms and Issues.” Chad Heatwole, Rita Bode, Nicholas Johnson, Christine Quinn, William Martens, Richard Moxley, Barbara Vickrey, David Victorson.  4/2011 at the 63rd AAN. Citation: Neurology, Volume 76, supplement 4, P06.275.
  • “Results from the PRISM-2 Study: Patient Reported Impact of Symptoms in Myotonic Dystrophy Type-2.”  Chad Heatwole, Nicholas Johnson, Rita Bode, Jeanne Dekdebrun; Nuran Dilek; James E. Hilbert; Elizabeth Luebbe; William Martens; Michael P. McDermott; Christine Quinn; Nan Rothrock; Charles Thornton; Barbara G. Vickrey; David Victorson; Richard T. Moxley.  IDMC-10.  Paris, France.  6/9/15.
  • “Myotonic Dystrophy Patient Question and Answer Session:  DM2: Symptoms Overview and Management Strategies.”  Myotonic Dystrophy Foundation Annual Conference.  Washington D.C.  9/17/16.
  • “DM2: Research Update.”  Myotonic Dystrophy Foundation Annual Conference.  Washington D.C.  9/17/16.
  • “DM2: Symptoms Overview and Management Strategies.”  Myotonic Dystrophy Foundation Annual Conference.  Washington D.C.  9/17/16.
  • “Myotonic Dystrophy: Lowering the Bar for Therapeutic Development in One of the World’s Most Clinically Diverse Diseases.”  Wake Forrest University Neurology Grand Rounds.  10/25/16.
  •  “Development and Validation of a Disease-Specific Patient-Reported Outcome Measure for DM2: The Myotonic Dystrophy Type-2 Health Index (MD2HI).”  Chad Heatwole, Nicholas Johnson, Jeanne Dekdebrun, Nuran Dilek, Elizabeth Luebbe, William Martens, Michael McDermott, Richard Moxley.  International Myotonic Dystrophy Meeting (IDMC-11).  San Francisco, CA.  9/7/2017.
  • “DM2: Disease Development and Symptom Management”, C. Heatwole, C. Engebrecht, S. Rosero. J. Weinstein, J. Seabury, A. Varma, N. Dilek. Oral session presented at: The Myotonic Dystrophy Foundation (MDF); September 7-9, 2023; Washington D.C., MD.
  • “Development of a Novel, Disease-Specific, Patient-Reported Outcome Measure; the Myotonic Dystrophy Type 2 Health Index (MD2HI)”. C. Engebrecht, S. Rosero, J. Seabury, A. Varma, J. Weinstein, S. Khosa, C. Heatwole. Poster session presented at: 2023 Neuromuscular Study Group Annual Scientific Meeting; September 22-24, 2023; Orlando, FL.
  •  “Development of a Disease-Specific Patient-Reported Outcome Measure in Myotonic Dystrophy Type 2: The Myotonic Dystrophy Type 2 Health Index (MD2HI)”. C. Engebrecht, S. Rosero, J. Weinstein, J. Seabury, A. Varma, S. Khosa, C. Shupe, C. Irwin, A. Brocht, C. Heatwole. Poster Presentation at the Muscular Dystrophy Association Conference; March 3-6, 2024; Orlando, FL.
  • “Development of a novel, disease-specific patient-reported outcome measure for myotonic dystrophy type 2: the MD2HI”.  C. Engebrecht, S. Rosero, J. Weinstein, J. Seabury, A. Varma, C. Shupe, C. Irwin, A. Brocht, N. Dilek, P. Kanagaiah, C. Heatwole. The 14th International Myotonic Dystrophy Consortium Meeting; April 9-13, 2024; Nijmegen, Netherlands.
  • “The Myotonic Dystrophy Type 2 Health Index (MD2HI): A Multifactorial, Disease-Specific, Patient-Reported Outcome Measure for Use in Therapeutic Trials”. C. Engebrecht, S. Rosero, J. Weinstein, J. Seabury, A. Varma, S. Khosa, C. Shupe, C. Irwin, A. Brocht, C. Heatwole.  The American Academy of Neurology 76th Annual Meeting; April 13-18, 2024; Denver, CO.
  • “The Myotonic Dystrophy Type 2 Health Index (MD2HI): Development and Validation of a Patient-reported Outcome Measure to Support Drug-Labeling Claims and Patient Monitoring”. C. Engebrecht, S. Rosero, J. Weinstein, J. Seabury, A. Varma, C. Shupe, C. Irwin, A. Brocht, N. Dilek, C. Heatwole. Poster session presented at: The Neuromuscular Study Group Conference; September 20-24, 2024; Tarrytown, NY.

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 MD2HI score (0-100) representing overall disease burden.

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