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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 2CDA+IFN | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cladribine and pegylated interpheron alpha-2a | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| To 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. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 6 months |
| To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Luis Escribano, MD, PhD | Contact | +34925269335 | lescribanom@sescam.jccm.es | |
| Iván Alvarez-Twose, MD | Contact | +34925269336 | ivana@sescam.jccm.es |
| Name | Affiliation | Role |
|---|---|---|
| Luis Escribano, MD, PhD | Instituto de Estudios de Mastocitosis de Castilla La Mancha | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle | Recruiting | Toledo | Toledo | 45071 | Spain |
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| ID | Term |
|---|---|
| D034721 | Mastocytosis, Systemic |
| D008415 | Mastocytosis |
| D000090362 | Mast Cell Activation Disorders |
| ID | Term |
|---|---|
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D017338 | Cladribine |
| ID | Term |
|---|---|
| D015762 | 2-Chloroadenosine |
| D000241 | Adenosine |
| D011684 | Purine Nucleosides |
| D011687 | Purines |
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Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy. |
| 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). | 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. | 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. | 6 months |
| 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. | 6 months |
| D007154 | Immune System Diseases |
| D006574 |
| Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D003839 | Deoxyadenosines |
| D003853 | Deoxyribonucleosides |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |