Muscle Dysfunction in Patients With Hematological Diseases Referred to Stem Cell Transplant

The Effect of Medical Treatment on Muscle Dysfunction and the Prognostic Role of Muscle Dysfunction at Critical Decision Points in Patients With Hematological Diseases Referred to Myeloablative Hematopoietic Stem Cell Transplant (HSCT), to Myeloablative HSCT With a "Reduced Toxicity Conditioning" Regime With Fludarabine and Treosulfane (FluTreo), or to Non-myeloablative HSCT. - A Prospective Observational Study.

PURPOSE: To investigate the effect of the disease and HSCT on muscle dysfunction and to investigate the prognostic role of muscle dysfunction at critical decision points in patients with hematological diseases referred to hematopoietic stem cell transplant (HSCT).

HSCT: Patients diagnosed with malignant hematological diseases who are referred to myeloablative HSCT, to a myeloablative "reduced toxicity conditioning" regime with Fludarabine and Treosulfane (FluTreo) or to non-myeloablative HSCT.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia; Myelodysplastic Syndromes; Hematologic Diseases; Chronic Lymphocytic Leukemia; Non Hodgkin Lymphoma; Chronic Myeloid Leukemia; Acute Lymphoid Leukemia; Stem Cell Transplant Complications
• Intervention / Treatment: Other: No intervention

Detailed Description

RATIONAL: Patients diagnosed with malignant hematological diseases undergoing HSCT are faced with poor prognosis. The treatment is demanding and associated with severe deconditioning potentially leading to worse prognostic outcomes. To what extend patients body composition at the point of referral to HSCT, as well as changes in body composition throughout the cancer continuum is associated with cancer outcomes is currently not well described, specifically if this should be part of standard clinical evaluation in order to optimize therapy-efficacy. Recent findings suggest that pathophysiological alterations in skeletal muscle mass and function can have significant implications for the risk of disease progression and long term prognosis.

• Study Type: Observational
• Enrollment (Estimated): 144

Contacts and Locations

• Study Contact: Name: Jan Christensen, post doc; Phone Number: 0045 22479075; Email: jan.christensen.02@regionh.dk
• Study Contact Backup: Name: Nina Høgdal, MSc; Phone Number: 0045 35450503; Email: nina.hoegdal@region.dk

Study Locations

• Denmark
  • Copenhagen, Denmark
    • Recruiting
    • Rigshospitalet
    • Contact: Jan Christensen, PhD
    • Contact: Nina Høgdal, MSc

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with malignant hematological diseases referred to myeloablative HSCT, myeloablative RTC-HSCT or non-myeloablative HSCT.

Description

Inclusion Criteria:

• Patients diagnosed with acute myelogenous leukaemia (AML), acute lymphatic leukaemia (ALL), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndrome (MDS), chronic lymphatic leukaemia (CLL), malignant lymphomas or multiple myeloma (MM) referred to myeloablative HSCT, myeloablative RTC-HSCT or non-myeloablative HSCT at the Department of Haematology, Rigshospitalet, Blegdamsvej.

Exclusion Criteria:

• age <18; pregnancy; physical or mental disabilities precluding test of muscle function; inability to read and understand Danish

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort 1 - Patients referred to myeloablative HSCT; These patients will undergo 5 assessments: a baseline-assessment 3-4 week prior to conditioning treatment, at discharge (in-patients) or at day +28 after stem cell infusion (out-patients) and follow-up assessments at 3 months, 6 months and 12 months.	Other: No intervention; No intervention
Cohort 2 - Patients referred to non myeloablative HSCT; These patients will undergo 5 assessments: a baseline-assessment 3-4 week prior to conditioning treatment, at discharge (in-patients) or at day +28 after stem cell infusion (out-patients) and follow-up assessments at 3 months, 6 months and 12 months.	Other: No intervention; No intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cohort 1 and Cohort 2 Medical treatment complications	Incidens rate of medical complications (mortality, re-hospitalization, infections, all cause disease relapse, chronic GVHD, return to work)	From baseline to 1 year follow-up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hospitalization duration	Total number days in hospital	From baseline to 1 year follow-up
Disease free survival	Risk of disease progression	From baseline to 1 year follow-up
Overall survival	Risk of mortality from any-cause	From baseline to 1 year follow-up
Change in whole body lean mass	Dual-energy X-ray Absorptiometry (DXA) scan	From baseline to 1 year follow-up
Change in appendicular lean mass	Dual-energy X-ray Absorptiometry (DXA) scan	From baseline to 1 year follow-up
Change in whole body fat percentage	Dual-energy X-ray Absorptiometry (DXA) scan	From baseline to 1 year follow-up
Change in visceral fat mass	Dual-energy X-ray Absorptiometry (DXA) scan	From baseline to 1 year follow-up
Change in bone mineral density	Dual-energy X-ray Absorptiometry (DXA) scan	From baseline to 1 year follow-up
Change in bone mineral content	Dual-energy X-ray Absorptiometry (DXA) scan	From baseline to 1 year follow-up
Change in walking capacity	Maximum 10 meter walking speed	From baseline to 1 year follow-up
Change in lower body physical function	30 seconds Sit-To- Stand test	From baseline to 1 year follow-up
Change in maximum leg power	Leg extensor power test	From baseline to 1 year follow-up
Change in inflammation markers	Blood values are registered from the patients hospital record in relation to assessments. C-reactive protein (CRP) and leucocytes are registered as they are inflammation markers	From baseline to 1 year follow-up
Change in creatinine and hemoglobin	Blood values are registered from the patients hospital record in relation to assessments. C-reactive protein (CRP) and leucocytes are registered as they have an influence on muscle strength	From baseline to 1 year follow-up
Change in body fat percentage, fat mass, fat-free mass, muscle mass and bone mass and total body water	Bioelectrical Impedance Analyzer	From baseline to 1 year follow-up
Change in health related quality of life	European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30 Version 3.0)	From baseline to 1 year follow-up
Change in psychological distress	Hospital Anxiety and Depression Scale (HADS) questionnaire	From baseline to 1 year follow-up
Change in sleep quality	Pittsburgh Sleep Quality Index (PSQI) questionnaire	From baseline to 1 year follow-up
Change in physical activity level	International Physical Activity Questionnaire (IPAQ) short form	From baseline to 1 year follow-up

Collaborators and Investigators

• Sponsor: Rigshospitalet, Denmark
• Investigators: Principal Investigator: Jan Christensen, post doc, Department of Occupational- and Physiotherapy, Rigshospitalet

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2023
• Primary Completion (Estimated): December 31, 2025
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: November 15, 2019
• First Submitted That Met QC Criteria: November 15, 2019
• First Posted (Actual): November 19, 2019

Study Record Updates

• Last Update Posted (Actual): April 11, 2024
• Last Update Submitted That Met QC Criteria: April 10, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Patient reported outcomes; Complications; Muscle strength; PRO; Physical function; Muscle mass; hematopoietic stem cell transplant
• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Disease Attributes; Bone Marrow Diseases; Myeloproliferative Disorders; Leukemia, B-Cell; Chronic Disease; Myelodysplastic Syndromes; Leukemia; Leukemia, Myeloid; Hematologic Diseases; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphocytic, Chronic, B-Cell; Leukemia, Lymphoid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: P-2019-223

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Not provided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No