About the study

The MAnaskin study evaluates the use of dupilumab in adults with indolent systemic mastocytosis involving the skin. The aim is to determine whether treatment with dupilumab can reduce disease symptoms and improve patients’ quality of life compared with standard symptomatic treatment.

The study is randomised, double-blind and placebo-controlled, allowing for an objective assessment of the treatment’s efficacy and safety.

What is systemic mastocytosis?

Systemic mastocytosis is a rare bone marrow neoplasm characterised by the abnormal accumulation and activation of mast cells. It is estimated to affect approximately 10–13 people per 100,000 population, meaning that around 5,000 individuals in Poland may be living with the disease.

In adults, the most common form is indolent systemic mastocytosis (ISM), a slowly progressing disease that may persist for many years. Although it often does not require haematological treatment, it can be associated with significant skin symptoms and manifestations related to the release of mast cell mediators.

Common symptoms include:

• itching and episodes of skin flushing;
• abdominal pain and diarrhoea;
• low blood pressure, headaches and concentration difficulties;
• osteopenia and osteoporosis.

Some patients are also at increased risk of anaphylactic reactions, particularly following insect stings or exposure to certain medications.

Why is a new treatment needed?

Current treatment of indolent systemic mastocytosis focuses mainly on symptom control. Antihistamines are commonly used to reduce symptoms, but they do not address the underlying mechanisms of the disease.

Various therapies, including tyrosine kinase inhibitors and biological agents, have been investigated. However, no targeted treatment has yet demonstrated a clear and consistent improvement in skin symptoms and quality of life in patients with ISM.

Why dupilumab?

Cytokines IL-4 and IL-13 play an important role in the mechanisms involved in mastocytosis. Dupilumab is a monoclonal antibody that targets the receptor shared by these cytokines.

The drug is already used in the treatment of several inflammatory conditions, including atopic dermatitis, severe asthma and chronic rhinosinusitis with nasal polyps. The MAnaskin study evaluates its potential benefits in patients with indolent systemic mastocytosis.

Study design

The study has been approved by the Ethics Committee of the Medical University of Gdańsk. Participation is voluntary and eligibility is assessed according to predefined protocol criteria.

Treatment allocation is performed randomly (randomisation), and neither participants nor investigators know which treatment is being administered at a given time (double-blind design).

Centre experience

The Department of Allergology at the Medical University of Gdańsk hosts a reference centre of the European Competence Network on Mastocytosis (ECNM). Approximately 500 patients from across Poland are currently under its care, representing one of the largest national cohorts included in the European registry of this disease.

Project objectives

The project aims to evaluate whether dupilumab can reduce skin lesions, alleviate symptoms associated with allergy and anaphylaxis, and improve quality of life compared with standard antihistamine treatment. The assessment includes, among other measures, serum tryptase levels and disease status following treatment.

Study population

Eligibility is always assessed individually on the basis of medical documentation and the study protocol criteria.

Organisational information

The project is funded by the Polish state budget through the Medical Research Agency (ABM). It was selected for funding under the second edition of the Head-to-Head Non-Commercial Clinical Trials and Research Experiments Competition (call number ABM/2023/1).

Project duration: 01 August 2023 – 31 July 2029.