| life: Within the past | |
|---|---|
| Section A: background | |
| Example Questions | Response type |
| Which country are you based in?* | Single Select |
| What is your age? Please select from the appropriate range below.* | Single Select |
| For how many people with MLD are you, or have you been the primary caregiver?* | Numerical |
| For how long have you been the primary caregiver for this person? | Numerical |
| Section B: pedsql general core scales | |
| Pediatric Quality of Life Inventory™ (PedsQL™) by Varni JW1 | |
| Section C: symptom burden | |
| Which of these answers comes closest to describing your child’s quality of life: At diagnosis?2 | Scaler (1–5) |
| Which of these answers comes closest to describing your child’s quality of life: Within the past 4 weeks? 2 | Scaler (1–5) |
| In the past 4 weeks, to what extent did any of the following physical symptoms impact your child as a result of their MLD: difficulty walking or crawling6 | Scaler (1–5) |
| In the past 4 weeks, to what extent did any of the following physical symptoms impact your child as a result of their MLD: Breathing/ respiratory problems6 | Scaler (1–5) |
| During the past 4 weeks, to what extent did your child’s condition interfere with his/her social activities with family, friends, neighbors, or groups?3 | Scaler (1–5) |
| During the past 4 weeks, to what extent did your child’s condition interfere with his/her school attendance? | Scaler (1–5) |
| Section D: treatment burden | |
| Did your child’s disease progression cause them to be ineligible for a transplant?* | Yes/No |
| What kind of donor was the transplant done with? | Single Select |
| Was conditioning used in preparation for your child’s Stem Cell Transplant? | Yes/No |
| What kind of conditioning was used? | Open-end |
| Section E: time investment | |
| When you think back on the following time periods, how many times did you and your child go to the hospital (inpatient) for his/her MLD, and how many total days did you stay there? If you are unsure, your best estimate will do: Since Diagnosis | Numerical |
| When you think back on the following time periods, how many times did you and your child go to the hospital (inpatient) for his/her MLD, and how many total days did you stay there? If you are unsure, your best estimate will do: Within the Past 12 Months* | Numerical |
| When you think back on the following time periods, how many times did you and your child go to the hospital (inpatient) for his/her MLD, and how many total days did you stay there? If you are unsure, your best estimate will do: Within the Past 4 Weeks* | Numerical |
| Is this number of days in the past month a typical number of days spent at the hospital for you and your child? | Yes/No |
| What was the reason for the hospitalization(s) within the past 4 weeks? | Open-end |
| How many days on average did you stay at the hospital for each hospitalization? | Numerical |
| Section F: social, emotional, and psychological burden | |
| Has your social life changed since your child was diagnosed? Please indicate to what extent you agree with each of these statements: I am as active socially as I had been before my child was diagnosed* | Scaler (1–7) |
| Has your social life changed since your child was diagnosed? Please indicate to what extent you agree with each of these statements: I miss many of my leisure activities that I used to enjoy before my child was diagnosed* | Scaler (1–7) |
| These questions are about how you felt and how things were with you during the past 4 weeks. For each question, please give the one answer that comes closest to the way you were feeling. How much of the time during the past 4 weeks: Did you feel overwhelmed?3* | Scaler (1–6) |
| These questions are about how you felt and how things were with you during the past 4 weeks. For each question, please give the one answer that comes closest to the way you were feeling. How much of the time during the past 4 weeks: Did you feel calm and peaceful? 3* | Scaler (1–6) |
| During the past 4 weeks, to what extent did your emotional state interfere with your social activities with family, friends, neighbors, or groups?3 | Scaler (1–5) |
| Within the past 4 weeks, in general, how would you rate your child’s mood?2 | Scaler (1–5) |
| Section G: financial and professional impact | |
| During the past 4 weeks, did you have any of the following problems with your work or other regular daily activities: Cut down the amount of time you spent on work or other activities3* | Yes/No |
| During the past 4 weeks, did you have any of the following problems with your work or other regular daily activities: Experienced work problems/difficulties* | Yes/No |
| Did you or your spouse/partner have to miss work as a result of your child’s condition?* | Yes/No |
| When you think back, how many days did you and your spouse/partner have to miss work due to MLD? If you are unsure, your best estimate will do: Since Diagnosis | Numerical |
| When you think back, how many days did you and your spouse/partner have to miss work due to MLD? If you are unsure, your best estimate will do: Within the Past 12 Months | Numerical |
| When you think back, how many days did you and your spouse/partner have to miss work due to MLD? If you are unsure, your best estimate will do: Within the Past 4 Weeks* | Numerical |
| Section H: demographics and closing | |
| Which of the following best describes your relationship status? | Single Select |
| What is the highest level of education that you have attained? | Single Select |
| Through which of the following do you have your primary form of health insurance coverage? | Single Select |
| Which of the following best represents your annual family income (before taxes)? | Single Select |
*Asterisks indicate questions included in manuscript
1. Licensed: Varni JW, Seid M, Kurtin PS. PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations. Med Care. 2001;39(8):800-812.
2.Questions adapted from PROMIS: Gruber-Baldini AL, Velozo C, Romero S, Shulman LM. Validation of the PROMIS® measures of self-efficacy for managing chronic conditions. Qual Life Res. 2017;26(7):1915-1924.
3.Questions adapted from SF-36: Brazier JE, Harper R, Jones NM, et al. Validating the SF-36 health survey questionnaire: new outcome measure for primary care. BMJ. 1992;305(6846):160-164.
4.Questions adapted from Neuro-QoL: Salsman JM, Victorson D, Choi SW, et al. Development and validation of the positive affect and well-being scale for the neurology quality of life (Neuro-QOL) measurement system. Qual Life Res. 2013;22(9):2569-2580.
5.Questions adapted from IMPA: Brown TM, Martin S, Fehnel SE, Deal LS. Development of the Impact of Juvenile Metachromatic Leukodystrophy on Physical Activities scale. J Patient Rep Outcomes. 2017;2(1):15.
6.Questions adapted from Eichler, et al. 2016: Eichler FS, Cox TM, Crombez E, Dali CÍ, Kohlschütter A. Metachromatic Leukodystrophy: An Assessment of Disease Burden. J Child Neurol. 2016;31(13):1457-1463.