The treatment of systemic mastocytosis has two main axes: * Control of mast cell activation symptoms and * The control of proliferation (accumulation) of mast cells. There is no standard treatment and no treatment has a marketing authorization for the treatment of monoclonal indolent mastocytosis.
Mastocytosis is an orphan disease related to the accumulation and / or the proliferation of abnormal mast cells in different tissues. In adults, a classic distinction is made between isolated cutaneous forms (10 to 15%) and systemic forms (85 to 90%). The treatment of systemic mastocytosis has two main axes: * Control of mast cell activation symptoms and * The control of proliferation (accumulation) of mast cells. There is no standard treatment and no treatment has a marketing authorization for the treatment of monoclonal indolent mastocytosis.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Patients will be treated by hydroxychloroquine at a dose of 6 to 6.5mg/kg/day during 12 month
Larrey Hospital - Toulouse University Hospital
Toulouse, France
Change of mast cell activation symptoms
The primary endpoint of this study is the change of mast cell activation symptoms as pruritus between the start of treatment and 12 months later. Skin pruritus will be assessed by the visual analogue scale from 0 to 10 at each visit.
Time frame: 12 month
Change of mast cell activation symptoms
The primary endpoint of this study is the change of mast cell activation symptoms as flushes between the start of treatment and 12 months later. The skin flush will be evaluated according to the absolute number of flushes / week at each visit
Time frame: 12 month
Difference on mast cell burden - serum tryptase level
The difference on mast cell burden between the start of treatment and 12 months later will be evaluated by variation of the level serum tryptase l expressed in μg / L.
Time frame: 12 month
Difference on skin mast cell burden - mast cells/mm²
The difference on mast cell burden between the start of treatment and 12 months later will be assessed by variation of the number of mast cells / mm² identified on the skin biopsies.
Time frame: 12 month
Difference of mast cell activation symptoms : diarrhea
The difference of diarrhea between the start of treatment and 12 months later evaluated by the absolute number of stools / day for diarrhea
Time frame: 12 month
Difference of mast cell activation symptoms : pollakiuria
The difference of pollakiuria between the start of treatment and 12 months later assessed by the absolute number of urinations / day for pollakiuria.
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Time frame: 12 month
Difference of mast cell activation symptoms : arthralgia
The difference of arthralgia between the start of treatment and 12 months later evaluated by the absolute number of painful joints / day and the intensity of joint pain assessed by the visual analogue scale from 0 to 10 for arthralgia.
Time frame: 12 month
Difference of mast cell activation symptoms : discomfort
The difference of discomfort between the start of treatment and 12 months later evaluated by the absolute number of faintness / week
Time frame: 12 month
The safety of hydroxychloroquine treatment.
The safety of hydroxychloroquine treatment will be done by evaluation of adverse events
Time frame: 12 month
effectiveness of treatment
The correlation between the efficacy of treatment with the hydroxychloroquine and level of serum HCQ will be performed by the Bland-Altman test.
Time frame: 12 month