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

Study Overview
=================
Brief Summary
-----------------
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.

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.

Official Title
-----------------
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.

Conditions
-----------------
Hematologic Diseases, Chronic Myeloid Leukemia, Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Non Hodgkin Lymphoma, Stem Cell Transplant Complications, Chronic Lymphocytic Leukemia

Intervention / Treatment
-----------------
* Other: No intervention


Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cohort 1 - Patients referred to myeloablative HSCT<br>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<br>* No intervention<br>|
| Cohort 2 - Patients referred to non myeloablative HSCT<br>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<br>* No intervention<br>|

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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Muscle mass, Muscle strength, Complications, Patient reported outcomes, PRO, Physical function, hematopoietic stem cell transplant