RATIONALE: Thalidomide may stop the growth of systemic mastocytosis by blocking blood flow to the disease. PURPOSE: This phase II trial is studying how well thalidomide works in treating patients with relapsed or progressive systemic mastocytosis.
Name: thalidomide
Type: DrugThalidomide
Allocation: Non-Randomized
Single Group Assignment
There is one SNP
DISEASE CHARACTERISTICS: - Diagnosis of systemic mastocytosis - Aggressive or borderline (smoldering) disease (in first line or more) - Relapsed or progressive disease - Measurable or evaluable disease - Presence of c-Kit D816V mutation in the skin, spine, or infiltrated organs - No nonsymptomatic mastocytosis PATIENT CHARACTERISTICS: - Life expectancy > 3 months - Not pregnant or nursing - Negative pregnancy test - Fertile patients must use effective contraception for 1 month prior to, during, and until first menstrual cycle after completion of study treatment - Bilirubin < 2 times normal (unrelated to disease) - Liver enzymes < 2 times normal (unrelated to disease) - Creatinine ≤ 300 mmol/L - No central or peripheral neuropathy leading to psychiatric concerns - No HIV positivity - No active infection or other serious underlying illness that would preclude treatment - No history of thromboembolism or deep vein thrombosis - No geographical, social, or psychological reasons preventing medical monitoring PRIOR CONCURRENT THERAPY: - More than 4 weeks since prior antitumor therapy (e.g., chemotherapy, radiotherapy) - No other concurrent treatment specific for this disease - No concurrent participation in another experimental drug trial DISEASE CHARACTERISTICS: - Diagnosis of systemic mastocytosis - Aggressive or borderline (smoldering) disease (in first line or more) - Relapsed or progressive disease - Measurable or evaluable disease - Presence of c-Kit D816V mutation in the skin, spine, or infiltrated organs - No nonsymptomatic mastocytosis PATIENT CHARACTERISTICS: - Life expectancy > 3 months - Not pregnant or nursing - Negative pregnancy test - Fertile patients must use effective contraception for 1 month prior to, during, and until first menstrual cycle after completion of study treatment - Bilirubin < 2 times normal (unrelated to disease) - Liver enzymes < 2 times normal (unrelated to disease) - Creatinine ≤ 300 mmol/L - No central or peripheral neuropathy leading to psychiatric concerns - No HIV positivity - No active infection or other serious underlying illness that would preclude treatment - No history of thromboembolism or deep vein thrombosis - No geographical, social, or psychological reasons preventing medical monitoring PRIOR CONCURRENT THERAPY: - More than 4 weeks since prior antitumor therapy (e.g., chemotherapy, radiotherapy) - No other concurrent treatment specific for this disease - No concurrent participation in another experimental drug trial Non Neoplastic Condition Precancerous Condition Mastocytosis Precancerous Conditions Mastocytosis, Systemic Disease OBJECTIVES: Primary - Determine the objective response rate at 6 months in patients with systemic mastocytosis treated with thalidomide. --- D816V ---
DISEASE CHARACTERISTICS: - Diagnosis of systemic mastocytosis - Aggressive or borderline (smoldering) disease (in first line or more) - Relapsed or progressive disease - Measurable or evaluable disease - Presence of c-Kit D816V mutation in the skin, spine, or infiltrated organs - No nonsymptomatic mastocytosis PATIENT CHARACTERISTICS: - Life expectancy > 3 months - Not pregnant or nursing - Negative pregnancy test - Fertile patients must use effective contraception for 1 month prior to, during, and until first menstrual cycle after completion of study treatment - Bilirubin < 2 times normal (unrelated to disease) - Liver enzymes < 2 times normal (unrelated to disease) - Creatinine ≤ 300 mmol/L - No central or peripheral neuropathy leading to psychiatric concerns - No HIV positivity - No active infection or other serious underlying illness that would preclude treatment - No history of thromboembolism or deep vein thrombosis - No geographical, social, or psychological reasons preventing medical monitoring PRIOR CONCURRENT THERAPY: - More than 4 weeks since prior antitumor therapy (e.g., chemotherapy, radiotherapy) - No other concurrent treatment specific for this disease - No concurrent participation in another experimental drug trial DISEASE CHARACTERISTICS: - Diagnosis of systemic mastocytosis - Aggressive or borderline (smoldering) disease (in first line or more) - Relapsed or progressive disease - Measurable or evaluable disease - Presence of c-Kit D816V mutation in the skin, spine, or infiltrated organs - No nonsymptomatic mastocytosis PATIENT CHARACTERISTICS: - Life expectancy > 3 months - Not pregnant or nursing - Negative pregnancy test - Fertile patients must use effective contraception for 1 month prior to, during, and until first menstrual cycle after completion of study treatment - Bilirubin < 2 times normal (unrelated to disease) - Liver enzymes < 2 times normal (unrelated to disease) - Creatinine ≤ 300 mmol/L - No central or peripheral neuropathy leading to psychiatric concerns - No HIV positivity - No active infection or other serious underlying illness that would preclude treatment - No history of thromboembolism or deep vein thrombosis - No geographical, social, or psychological reasons preventing medical monitoring PRIOR CONCURRENT THERAPY: - More than 4 weeks since prior antitumor therapy (e.g., chemotherapy, radiotherapy) - No other concurrent treatment specific for this disease - No concurrent participation in another experimental drug trial Non Neoplastic Condition Precancerous Condition Mastocytosis Precancerous Conditions Mastocytosis, Systemic Disease OBJECTIVES: Primary - Determine the objective response rate at 6 months in patients with systemic mastocytosis treated with thalidomide. --- D816V --- --- D816V ---