Studying Changes in Physical Function Measures in Stem Cell Transplant Patients at Risk for Steroid Myopathy

Pilot Study Evaluating Changes in Physical Function Measures in Stem Cell Transplant Patients at Risk for Steroid Myopathy

Status

Terminated

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04521777

Enrollment

Registered

2020-08-21

Start date

2015-10-01

Completion date

2021-03-18

Last updated

2021-04-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acute Graft Versus Host Disease

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.

Interventions

BEHAVIORALExercise Intervention

Participate in an 8-week home-based strengthening and walking program

OTHERPhysical Performance Testing

Complete a physical function test

OTHERQuestionnaire Administration

Ancillary studies

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

SUPPORTIVE_CARE

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
6 Minutes Walk Test	Baseline, Day 14, Day 28 and Day 56	6 minute walk test (measured in meters) was conducted on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0, and Day 56 minus Day 0.
5 Times Sit to Stand (5xSTS)	Baseline, Day 14, Day 28 and Day 56	5xSTS was measured in seconds on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.
Brooke Scale for Myopathy	Baseline, Day 14, Day 28 and Day 56	Brooke Scale for myopathy (a score from 0 to 6, with 6 being the best score with full range of motion of the arms) was measured on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0
Modified Adult Myopathy Assessment Tool	Baseline, Day 14, Day 28 and Day 56	The modified Adult Myopathy Assessment Tool assigns a numerical score to a series of physical tasks (arm raise \[0 to 3\], sustained arm raise \[0 to 4\], single sit-to-stand \[0 to 3\], sustained hip flexion \[0 to 4\], and sustained knee extension \[0 to 4\]). The total score of all test components (0 to 18) is reported, with 18 as the highest score for muscle function and endurance.
Knee Extensors Strength	Baseline, Day 14, Day 28 and Day 56	Knee Extensors Strength (in pounds) was measured on Baseline, Day 14, Day 28 and Day 56 and measured the median change for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.
Hip Flexors Strength	Baseline, Day 14, Day 28 and Day 56	Hip Flexors Strength (in pounds) was measured on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.

Countries

United States

Participant flow

Recruitment details

Patients who were treated with high dose corticosteroid for Acute Graft Versus Host Disease at MD Anderson Cancer Center between October 2015 and April 2017.

Participants by arm

Arm	Count
All Participants In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.	23
Total	23

Arm

Count

All Participants

In this single-arm trial, patients who were started on high dose corticosteroids (methylprednisolone or equivalent, 2mg/kg/day) for suspected Acute Graft Versus Host Disease were enrolled on the trial. In order monitor for muscle weakness, serial physical function measures were obtained (baseline, Day 14 after enrollment, Day 28, and Day 56). All enrolled patients were also instructed on a home-based exercise program.

Total

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Death	2
Overall Study	Withdrawal by Subject	2

Baseline characteristics

Characteristic	All Participants
Age, Continuous	60 years
Body Mass Index	29 kg/m^2
Cancer Status Active	6 Participants
Cancer Status Refractory	2 Participants
Cancer Status Remission	15 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants
Graft Source Bone marrow	4 Participants
Graft Source Peripheral Blood	19 Participants
Primary Cancer Acute Lymphocytic Leukemia	2 Participants
Primary Cancer Acute Myeloid Leukemia	7 Participants
Primary Cancer B cell Lymphoma	3 Participants
Primary Cancer Chronic Lymphocytic Leukemia	2 Participants
Primary Cancer Chronic Myeloid Leukemia	4 Participants
Primary Cancer Myelodysplastic Syndrome	4 Participants
Primary Cancer Myelofibrosis	1 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants
Race (NIH/OMB) Asian	2 Participants
Race (NIH/OMB) Black or African American	0 Participants
Race (NIH/OMB) More than one race	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants
Race (NIH/OMB) White	21 Participants
Region of Enrollment United States	23 participants
Sex: Female, Male Female	9 Participants
Sex: Female, Male Male	14 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	3 / 24
other Total, other adverse events	0 / 24
serious Total, serious adverse events	0 / 24

Outcome results

Primary

5 Times Sit to Stand (5xSTS)

5xSTS was measured in seconds on Baseline, Day 14, Day 28 and Day 56 and measured the median changes for Day 14 minus Day 0, Day 28 minus Day 0 and Day 56 minus Day 0.