Clinical trial NCT06065007: Quality of Life and Disease-related Symptoms in Individuals With Systemic Mastocytosis
Official title: Quality of Life and Disease-related Symptoms in Individuals With Systemic Mastocytosis
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Mastocytosis, Systemic
Lead sponsor: Uppsala University
Sponsor class: OTHER
Collaborators: name: Karolinska Institutet
First posted: 2023-10-03
Enrollment: 400
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: CROSS_SECTIONAL
Sampling method: PROBABILITY_SAMPLE
Study population: All patients diagnosed with Systemic Masocytosis and meets the eligibility criteria, who are registered at one of the two existing mastocytosis centres in Sweden (Uppsala or Stockholm), or can be identified through a regional representative, will be asked to participate

Brief summary: 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: 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.

Eligibility criteria: 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

Primary outcomes:
measure: EORTC QLQ C-30
timeFrame: Day 1
measure: The Mastocytosis Quality of Life Questionnaire (MC-QoL)
timeFrame: Day 1
measure: The Hospital Anxiety and Depression Scale (HADS)
timeFrame: Day 1
measure: The Gastrointestinal Symptom Rating Scale (GSRS)
timeFrame: Day 1
measure: The Visceral Sensitivity Index (VSI)
timeFrame: Day 1
measure: The Brief Pain Inventory - Short form (BPI-SF)
timeFrame: day 1
measure: The Brunnsviken Brief Quality of life scale (BBQ)
timeFrame: Day 1
measure: Self Care of Chronic Illness Inventory
timeFrame: Day 1
Secondary outcomes:
Other outcomes:
Locations:
city: Stockholm
country: Sweden
facility: Karolinska Institutet
city: Uppsala
country: Sweden
facility: Uppsala University
Study officials:
affiliation: Uppsala University, Department of Public Health and Caring Sciences
name: Mariann Hedström, PhD
role: PRINCIPAL_INVESTIGATOR
References:
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