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Transfusional dependence has been associated closely and independently with low survival in patients with myelodysplastic syndrome (MDS), especially in patients at low risk according to IPSS. Treatment of patients with hydralazine + valproic acid as an alternative to treatment with 5-azacytidine has lower cost and possibly as effective with fewer side effects. The objective of this phase II study is to determine the effectiveness of combination therapy with hydralazine + Ac. Valproic compared with best supportive care. The investigators will select 42 patients per group, and after 14 weeks of treatment the investigators will study in both groups the hematological response (transfusion-dependent, hemoglobin, cytogenetics and morphology) and treatment safety (adverse reactions and vital signs) to 1 year after starting treatment. The concentration of hemoglobin, the number of transfusions, platelets, neutrophils and other continuous variables in both groups will be compared by Student t or Mann-Whitney, as appropriate. For comparison of cytogenetic and morphological response and other categorical variables between groups Chi square will be used. And within each group the investigators will compare each of these variables before and after treatment by t-test for paired data or Wilcoxon test.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| HIDRA/VPA | Experimental | In each cycle (30 days), Hydralazine 50mg tablets every 12 hours and Valproic 500mg tablets every 8 hours will be administrated orally to HYDRA / VPA group.Each patient will receive 6 cycles of hydralazine and valproic acid. |
|
| best supportive care (BSC) | Active Comparator | The support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BSC | Drug | The support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician. |
|
| Measure | Description | Time Frame |
|---|---|---|
| change in hemoglobin from baseline in patients who express an erythroid response. | Independence RBC transfusion requirement for ≥ 8 weeks (56 days) followed by treatment at any time. | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Erythroid response according to the criteria of the international working group of SMDs at 24 weeks | Number and type of administered blood transfusions, time between the last transfusion prior to inclusion in the study and the first transfusion after the 8 weeks of treatment. | 24 weeks |
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Inclusion Criteria:
Exclusion Criteria:
Patients who have any of these exclusion criteria may not be included in the trial:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Breno Moreno de Gusmao, MD | Contact | +34-945 007000 | breno.morenodegusmao@osakidetza.net |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Txagorritxu Hospital | Vitoria-Gasteiz | Alava | 01009 | Spain |
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| HIDRA/VPA | Drug | In each cycle (30 days), Hydralazine 50mg tablets every 12 hours and Valproic 500mg tablets every 8 hours will be administrated orally to HYDRA / VPA group.Each patient will receive 6 cycle. |
|
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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