Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

PROTOCOL OUTLINE: This is a multicenter study. Patients receive arginine butyrate IV over 6-14 hours on days 1-5 of weeks 1-4 and 7-10. Patients then receive maintenance arginine butyrate IV over 6-14 hours on days 1-4 of weeks 13, 15, 17, 19, 21, 23, and 25.

Patients who have no medical contraindications (e.g., paraspinal extramedullary hematopoiesis, hypertension, or poorly controlled congestive heart failure) may continue therapy. Patients receive arginine butyrate IV over 6-14 hours on days 1-4 of weeks 27, 29, 31, 33, 35, 37, and 39 and epoetin alfa intramuscularly (IM) or subcutaneously (SC) three times weekly on weeks 27-40.

Patients may continue to receive epoetin alfa IM or SC alone three times weekly on weeks 41-52. Patients with severe anemia (hemoglobin less than 7 g/dL) may receive epoetin alfa alone on weeks 1-12 before arginine butyrate induction therapy.

Patients who complete therapy at week 26 are followed every 2 weeks for 2 months. Patients who complete therapy at week 40 are followed monthly for 2 months.

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

• Diagnosis of thalassemia intermedia with hemoglobin no greater than 10 g/dL Two beta thalassemia mutations
• Must have undergone prior splenectomy or have no palpable spleen

--Prior/Concurrent Therapy--

• At least 3 months since prior red blood cell transfusion

--Patient Characteristics--

• Performance status: SWOG 0-2
• Hematopoietic: No severe iron overload or ferritin greater than 5,000 ng/mL
• Hepatic: Normal hepatic function No active hepatitis
• Renal: Normal renal function
• Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception Must be willing to have vascular access placed No viral disease No contraindication to study compliance