Quality of Life and Disease-related Symptoms in Individuals With Systemic Mastocytosis

Study Overview
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Brief Summary
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Systemic Mastocytosis is a rare and complex disease caused by accumulation of mast cells. The skin, bones, gastrointestinal tract, bone marrow and liver are the organs most often affected. Symptoms can vary greatly between patients. The study aims to describe the Swedish cohort's self-rated quality of life and levels of disease-related symptoms.

Detailed Description
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Systemic Mastocytosis (SM) is a rare and complex disease caused by accumulation of mast cells leading to release of mediator substances (e.g., cytokines, prostaglandins, histamine and tryptase). The skin, bones, gastrointestinal tract, bone marrow and liver are the organs most often affected. Symptoms vary between patients and can include e.g., allergic reactions with anaphylaxis, rashes, osteoporoses, cognitive difficulties, fatigue, depression, anxiety, nausea, vomiting, stomach pains and diarrhea. In this cross-sectional observational study, the aim is to include the Swedish cohort of persons diagnosed with SM to gather a wide range of information on self-rated health-related quality of life, gastrointestinal symptoms, pain, anxiety, depression and self-care.

Official Title
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Quality of Life and Disease-related Symptoms in Individuals With Systemic Mastocytosis

Conditions
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Mastocytosis, Systemic

Intervention / Treatment
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* Other: No intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Swedish-speaking individuals aged 18 years or older and diagnosed with indolent systemic mastocytosis (ISM) or advanced systemic mastocytosis (AdvSM), verified by a bone marrow biopsy Exclusion Criteria: An assessment by the treating physician that the individual has a cognitive impairment making participation impossible The patient has undergone an allogeneic stem cell transplant

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Swedish cohort of persons with Systemic Mastocytosis<br>We aim to include all individuals with a diagnosis of SM in Sweden, by identification via either one the two centers of excellence of mastocytosis or by a regional representative for the mastocytosis group in Sweden. | Other: No intervention<br>* This is a cross-sectional observational study, no intervention will be implemented or exposure studied. Data for outcomes of interest will be collected via questionnaires<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| EORTC QLQ C-30 | EORTC QLQ C-30 measures health related quality of life rated by individuals with malignancies and the general population. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus, a high score for a functional scale represents a high / healthy level of functioning, a high score for the global health status / QoL represents a high QoL, but a high score for a symptom scale / item represents a high level of symptomatology / problems | Day 1 | 
| The Mastocytosis Quality of Life Questionnaire (MC-QoL) | The Mastocytosis Quality of Life Questionnaire (MC-QoL), measures disease-specific quality of life items, has scores from 0 to 100, where higher scores indicate higher health-related quality-of-life impairment | Day 1 | 
| The Hospital Anxiety and Depression Scale (HADS) | The Hospital Anxiety and Depression Scale (HADS), 14 items, is a screening tool for anxiety and depression in non-psychiatric clinical populations. There are 7 items for depression and 7 items for anxiety. Each item on the questionnaire is scored from 0-3 and this means that a person can score between 0 and 21 for either anxiety or depression. A higher score indicates a higher level of distress. | Day 1 | 
| The Gastrointestinal Symptom Rating Scale (GSRS) | The Gastrointestinal Symptom Rating Scale (GSRS), 15 items. This instrument measures The Gastrointestinal Symptom Rating Scale (GSRS). This instrument measures gastrointestinal symptoms in individuals with irritable bowel syndrome, gastric or duodenal ulcers and dyspepsia. The questionnaire, which contains 15 items, 6 subscales with total score between 19 to 133. Higher scores indicates more symptoms. | Day 1 | 
| The Visceral Sensitivity Index (VSI) | The Visceral Sensitivity Index (VSI), 15 items, measures psychosocial consequences and worries of gastrointestinal symptoms. Each item scores 0-6 and a higher score indicates greater GI symptom-specific anxiety. | Day 1 | 
| The Brief Pain Inventory - Short form (BPI-SF) | The Brief Pain Inventory - Short form (BPI-SF), 9 items, yields a rating of levels of pain during a 24-hour period, and a description of the nature of that pain. The instrument also contains items on the impact (if any) of the pain of daily life. Each rating scale is bounded by the words no pain at the 0 end and pain as bad as you can imagine at the other. Using similar scales of 0 to 10, patients are also asked to rate the extent to which their pain interferes with 7 quality-of-life domains that include general activity, walking, mood, sleep, work, relations with other persons, and enjoyment of life. These scales are bounded by the words does not interfere and interferes completely: Higher scores indicates higher levels of pain/Higher impact of pain on daily life. | day 1 | 
| The Brunnsviken Brief Quality of life scale (BBQ) | The Brunnsviken Brief Quality of life scale (BBQ), 12 items, has 4-graded response alternatives. The scale measures the overall self-experienced quality of life. The Satisfaction and Importance rating for each life area is multiplied and summed for a total quality of life score ((items 1 * 2) + (items 3 * 4) and so forth). Higher scores indicate higher levels of subjective QoL. | Day 1 | 
| Self Care of Chronic Illness Inventory | Self Care of Chronic Illness Inventory, 29 items, measures self-care behaviors in persons with chronic illnesses. The scale measures three aspects of self care; Self-Care Maintenance, Self-Care Monitoring (or Symptom Perception), and Self-Care Management. Scores on each scale are standardized to range from 0 to100 with higher scores indicating better self-care | Day 1 |