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.
Name: Cladribine and pegylated interpheron alpha-2a
Description: 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.Type: Drug2CDA+IFN
Description: Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.
Measure: To evaluate the effect of therapy on bone marrow mast cell infiltration. Time: 6 monthsDescription: Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy.
Measure: To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis. Time: 6 monthsDescription: Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy.
Measure: To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis). Time: 6 monthsDescription: Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE).
Measure: To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a. Time: 6 monthsDescription: Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry.
Measure: To evaluate the effect of therapy on mastocytosis skin lesions. Time: 6 mothsDescription: Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography.
Measure: To evaluate the effect of therapy on mastocytosis-related organomegalies. Time: 6 monthsDescription: Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography.
Measure: To evaluate the effect of therapy on mastocytosis-related bone alterations. Time: 6 monthsSingle Group Assignment
There is one SNP
Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.. Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis 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. --- D816V ---
Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.. Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis 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. --- D816V --- --- D816V ---
- Diagnosis of advanced systemic mastocytosis (aggressive systemic mastocytosis or proggressing systemic mastocytosis) with D816V or other exon 17 KIT mutations. --- D816V ---