The aim of this study is to evaluate the efficacy in terms of clinical and biological response rates of Cladribine plus Pegylated Interpheron alpha-2a therapy in patients with advanced systemic mastocytosis carrying D816V or other exon 17 KIT mutations.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Cladribine (0.07 mg/Kg/day) s.c for 5 consecutive days each month for a total of 6 months.Cladribine daily doses could be increased up to 0.14 mg/Kg in the fourth, fifth and sixth cycles of therapy if no objetive response is achieved after the third cycle. Pegylated Interpheron alpha-2a (1 mcgr/Kg) s.c weekly for a total of 6 months.
Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle
Toledo, Toledo, Spain
RECRUITINGTo evaluate the effect of therapy on bone marrow mast cell infiltration.
Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.
Time frame: 6 months
To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.
Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy.
Time frame: 6 months
To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis).
Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy.
Time frame: 6 months
To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a.
Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE).
Time frame: 6 months
To evaluate the effect of therapy on mastocytosis skin lesions.
Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry.
Time frame: 6 moths
To evaluate the effect of therapy on mastocytosis-related organomegalies.
Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography.
Time frame: 6 months
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To evaluate the effect of therapy on mastocytosis-related bone alterations.
Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography.
Time frame: 6 months