Clinical trial NCT04521777: Studying Changes in Physical Function Measures in Stem Cell Transplant Patients at Risk for Steroid Myopathy
Official title: Pilot Study Evaluating Changes in Physical Function Measures in Stem Cell Transplant Patients at Risk for Steroid Myopathy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Acute Graft Versus Host Disease
Lead sponsor: M.D. Anderson Cancer Center
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2020-08-21
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: This trial investigates the changes in physical and functional tests over time in patients with suspected acute graft versus host disease (from a hematologic stem cell transplant) who started treatment with corticosteroids. Sometimes the transplanted cells from a donor can attack the body's normal cells (called graft-versus-host disease). Steroids are used to treat suspected graft-versus-host disease. Steroid myopathy (muscle weakness and fatigue) is a significant side effect of high dose steroid therapy, and can impair activities of daily of life. The goal of this trial is to learn how patients' physical activities and functions change over time while on GVHD-steroid treatment.

Detailed description: PRIMARY OBJECTIVE:

I. To perform a pilot study to estimate the change in six physical and functional tests over time in patients with suspected acute graft-versus-host disease (GVHD) who have been initiated on treatment with corticosteroids.

SECONDARY OBJECTIVES:

I. To describe and potentially define steroid myopathy by following the patterns of the muscle loss and functional impairment in this population of patients.

II. To follow steroid myopathy and describe its incidence, its severity, and the impact on non-relapse mortality in a homogeneous population of patients with suspected acute graft-versus-host disease (GVHD) who have been initiated on treatment with corticosteroids at a quaternary institution in the inpatient and outpatient setting.

III. To estimate the adherence to an intermittently supervised exercise program in hematopoietic stem cell transplantation (HSCT) patients who are at risk of developing steroid myopathy.

OUTLINE:

Patients complete a physical function test over 15 minutes at baseline, and at days 14, 28, and 56. Patients also participate in an 8-week home-based strengthening and walking program consisting of a strengthening/resistance program for 30 minutes, 3 times a week, and walking program for 20-30 minutes at least 3 times a week.

Eligibility criteria: Inclusion Criteria:

* Participants are willing and able to give written informed consent and to comply with all of the study visits and procedures
* Age \>= 55 years, or with a Sorror co-morbidity index of \>= 3
* Post allogenic hematopoietic stem cell transplantation using bone marrow, peripheral blood or cord blood; or after pre planned donor lymphocyte infusion
* Presumptive diagnosis of acute GVHD necessitating high-dose corticosteroid treatment (with an approximate starting dose of methylprednisolone equivalent of 2 mg/kg/day)
* Within 5 days of receiving corticosteroid treatment
* ECOG (Eastern Cooperative Oncology Group) performance status of 0, 1, or 2 or equivalent Karnofsky score of 60 or higher
* The patient is referred to the study by their stem cell transplant attending physician

Exclusion Criteria:

* Non-English speaking
* Underlying unstable cardiac or pulmonary disease in the opinion of the investigator that limits participant involvement in exercise
* Has a pre-transplant echocardiogram with ejection fraction \< 45%
* Requires supplemental oxygen to maintain oxygen (O2) saturation \> 92%
* Musculoskeletal injury that precludes participation in an exercise program
* Inability to participate in a structured exercise program
* Patients for whom the physician feels is unsafe for an exercise program
* Platelets equal to or less than 10,000 or evidence of active bleeding
* Patients who are unable to understand or follow through with the exercise program

Primary outcomes:
measure: 6 Minutes Walk Test
timeFrame: Baseline, Day 14, Day 28 and Day 56
measure: 5 Times Sit to Stand (5xSTS)
timeFrame: Baseline, Day 14, Day 28 and Day 56
measure: Brooke Scale for Myopathy
timeFrame: Baseline, Day 14, Day 28 and Day 56
measure: Modified Adult Myopathy Assessment Tool
timeFrame: Baseline, Day 14, Day 28 and Day 56
measure: Knee Extensors Strength
timeFrame: Baseline, Day 14, Day 28 and Day 56
measure: Hip Flexors Strength
timeFrame: Baseline, Day 14, Day 28 and Day 56
Secondary outcomes:
Other outcomes:
Locations:
city: Houston
country: United States
facility: M D Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: An T Ngo-Huang
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ngo-Huang A, Yadav R, Bansal S, Williams J, Wu J, Fu JB, Alousi AM, Bruera E. An Exploratory Study on Physical Function in Stem Cell Transplant Patients Undergoing Corticosteroid Treatment for Acute Graft-Versus-Host-Disease. Am J Phys Med Rehabil. 2021 Apr 1;100(4):402-406. doi: 10.1097/PHM.0000000000001660.