The Burden of Primary Caregivers of Spinal Muscular Atrophy Patients and Their Needs

NCT04228718 | Completed

• 1 other identifier: 09.2019.728
• Study Type: observational
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess carer burden, needs, and expectations of Spinal Muscular Atrophy Parents

Conditions

Spinal Muscular Atrophy; Caregiver Burden

Keywords

Spinal Muscular Atrophy; Caregiver Burden; Disabled persons; Caregivers

Outcome Measures

Primary Outcomes (2)

• Zarit Burden Interview Scale

  It is a questionnaire consisting of 22 items. A minimum score of 0 and a maximum score of 88 can be obtained. The higher the scale score, the higher the difficulty experienced.

  Day 0

• Family Needs Survey (FNS)

  It is a questionnaire consisting of 35 items prepared to determine the requirements. In the FNS survey; The points given to the items can be directly collected and the amount of family needs can be determined over the total score. The lowest score that can be obtained from FNS is 29 and the highest score is 87. As the scores obtained from the scale and subscales increase, it can be said that the families' level of necessity increases.

  Day 0

Secondary Outcomes (2)

• Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)

  Day 0

• Hammersmith Functional Motor Scale Expanded (HMFSE)

  Day 0

Interventions

Zarit Burden Interview Scale BEHAVIORAL

It is a questionnaire consisting of 22 items. A minimum score of 0 and a maximum score of 88 can be obtained. The higher the scale score, the higher the difficulty experienced.

Family Needs Survey (FNS) BEHAVIORAL

It is a questionnaire consisting of 35 items prepared to determine the requirements. In the FNS survey; The points given to the items can be directly collected and the amount of family needs can be determined over the total score. The lowest score that can be obtained from FNS is 29 and the highest score is 87. As the scores obtained from the scale and subscales increase, it can be said that the families' level of necessity increases.

Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) OTHER

CHOP-INTEND uses a scale of 0-64 points. Higher scores indicate better motor function. It is used to reliably measure natural motor function decline in infants with SMA Type 1.

Hammersmith Functional Motor Scale Expanded (HMFSE) OTHER

It is an extended version of the 20-item HFMS. Based on another scale known as GMFM, 13 items were added and expanded HFMS to include these elements. Therefore, HFMSE is a scale used to evaluate motor functions in types 2 and 3 SMA. It contains 33 items with a total score of 66 on a scale of 0, 1, 2.

Eligibility Criteria

• Age: 1 Day - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Patients with SMA type 1, type 2 or type 3 between the ages of 0-18 and their caregivers.

You may qualify if:

• Patients with spinal muscular atrophy at between the ages of 0-18
• Agree to participate in the study

You may not qualify if:

• Refuse to participate in the study

Sponsors & Collaborators

• Marmara University: lead

Study Sites (1)

Marmara University School of Medicine Department of Physical Medicine and Rehabilitation

Istanbul, 34890, Turkey (Türkiye)

Location

Related Publications (6)

• Flunt D, Andreadis N, Menadue C, Welsh AW. Clinical commentary: obstetric and respiratory management of pregnancy with severe spinal muscular atrophy. Obstet Gynecol Int. 2009;2009:942301. doi: 10.1155/2009/942301. Epub 2009 May 19.

  PMID: 19960049 BACKGROUND

• Pearn J. Incidence, prevalence, and gene frequency studies of chronic childhood spinal muscular atrophy. J Med Genet. 1978 Dec;15(6):409-13. doi: 10.1136/jmg.15.6.409.

  PMID: 745211 BACKGROUND

• Strober JB, Tennekoon GI. Progressive spinal muscular atrophies. J Child Neurol. 1999 Nov;14(11):691-5. doi: 10.1177/088307389901401101.

  PMID: 10593543 BACKGROUND

• Wirth B, Brichta L, Schrank B, Lochmuller H, Blick S, Baasner A, Heller R. Mildly affected patients with spinal muscular atrophy are partially protected by an increased SMN2 copy number. Hum Genet. 2006 May;119(4):422-8. doi: 10.1007/s00439-006-0156-7. Epub 2006 Mar 1.

  PMID: 16508748 BACKGROUND

• Ho HM, Tseng YH, Hsin YM, Chou FH, Lin WT. Living with illness and self-transcendence: the lived experience of patients with spinal muscular atrophy. J Adv Nurs. 2016 Nov;72(11):2695-2705. doi: 10.1111/jan.13042. Epub 2016 Jun 30.

  PMID: 27293032 BACKGROUND

• Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK, Sproule DM, Kang PB, Foley AR, Yang ML, Martens WB, Oskoui M, Glanzman AM, Flickinger J, Montes J, Dunaway S, O'Hagen J, Quigley J, Riley S, Benton M, Ryan PA, Montgomery M, Marra J, Gooch C, De Vivo DC. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014 Aug 26;83(9):810-7. doi: 10.1212/WNL.0000000000000741. Epub 2014 Jul 30.

  PMID: 25080519 BACKGROUND

MeSH Terms

Conditions

Muscular Atrophy, Spinal; Caregiver Burden

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases; Stress, Psychological; Behavioral Symptoms; Behavior

Study Officials

• Naime Evrim Karadag Saygi, Prof

  Department of PM&R, Marmara University School of Medicine

  STUDY DIRECTOR

• Ayca Evkaya, Res. Asst.

  Department of Physiotherapy and Rehabilitation, Maltepe University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 12, 2020
• First Posted: January 14, 2020
• Study Start: July 1, 2019
• Primary Completion: February 28, 2020
• Study Completion: May 1, 2020
• Last Updated: May 14, 2020

Record last verified: 2020-01

Locations

TU (1)