eltrombopag in second line adult primary immune thrombosytopenia

Conditions: Primacy Immune Thrombocytopenia

Summary: a clinical study to evaluate the efficacy of a drug called eltrombopag in adult patients affected of primary immune thrombocytopenia as first treatment to address the disease.

this is a prospective, multicenter phase ii trial designed to evaluate the activity of eltrombopag second line treatment in adult patients with primary immune thrombocytopenia (itp) not responsive or in relapse after a full first line steroids treatment (prednisone or dexamethasone) ± intravenous immune globulin (ivig).

Eligibility criteria:
inclusion criteria: 1. diagnosis of primary itp; 2. age ≥ 18 years; 3. patients are in the new diagnosis or persistent phase of disease (i.e. within month 12 from diagnosis); 4. patients not responsive or in relapse after a full course of steroid therapy (prednisone 1 mg/kg/d for at least 28 days or 3 cycles of dexamethasone 40 mg/day for consecutive 4 days, according to the gimema itp0207 trial) ± ivig; 5. patients have a platelet count \< 10 x 109/l documented in a single blood cell count; 6. patients have bleeding symptoms and a platelet count \> 10 \< 30 x 109/l documented in a single blood cell count; 7. patients have no bleeding symptoms and a platelet count \>10 \< 30 x 109/l in at least 2 blood cell counts at 3 days interval in the week preceding the enrollment with the last count at the day of enrollment; 8. patients have corticosteroids or ivig dependence (the need for ongoing or repeated doses administration of corticosteroids or ivig to maintain a platelet count ≥ 30 x 109/l and/or to avoid bleeding; 9. written informed consent obtained from the subject; 10. men with a female partner of childbearing potential must have either had a prior vasectomy or agree to use effective contraception from time of enrollment until 6 months after the last dose of study treatment; 11. female subjects of non-childbearing potential may be enrolled in the study; for this study population, non-childbearing potential is defined as current tubal ligation, hysterectomy, ovariectomy or post-menopause; or 12. female subjects of childbearing potential may be enrolled in the study, if the subject has practiced adequate contraception for 30 days prior to start of eltrombopag, has a negative pregnancy test within 14 days of first dose of eltrombopag, and has agreed to continue adequate contraception during the entire treatment period and for 6 months after completion of the treatment. exclusion criteria: 1. diagnosis of secondary itp. as far as patients with immune thrombocytopenia and antiphospholipid antibodies positivity, individuals without a previous thromboembolic event are excluded only if lupus anticoagulant (lac) is associated with the presence of anticardiolipin (acl) and aβ2-glycoprotein i (aβ2gpi) antibodies; 2. previous treatment with other anti-itp second line therapies (i.e. rituximab, azathioprine, cyclosporin-a or other); only patients with a previous full course of steroid (see inclusion criteria for definition) ± ivig are admitted to the study; 3. previous treatment with any tpo-r agonists; 4. patients have life threatening bleeding complications; 5. patients had deep venous thrombosis (dvt) or arterial thrombosis in the 3 months preceding the enrollment; 6. patients are hiv, hcv, hbsag positive; 7. patients with hepatic impairment (i.e. mild, moderate or severe hepatic impairment (child-pugh score \> 6); 8. patients have a well established liver disease that represents a contraindication for the use of eltrombopag; 9. patients are unable to respect the 4-hour interval between eltrombopag and other medications (e.g. antacids), calcium-rich foods (e.g. dairy products and calcium fortified juices), or supplements containing polyvalent cations such as iron, calcium, aluminium, magnesium, selenium, and zinc; 10. patients are unable to stop medications that are known to cause a drug-drug interaction with eltrombopag; 11. subjects meeting any of the following criteria must not be enrolled in an eltrombopag study: * lactating female. * history of another malignancy. exception: subjects who have been disease-free for 5 years, or subjects with a history of completely resected non-melanoma skin cancer or successfully treated in situ carcinoma are eligible. * any serious and/or unstable pre-existing medical, psychiatric disorder, or other conditions that could interfere with subject's safety, obtaining informed consent or compliance with the study procedures. * hormone replacement therapy. subjects must discontinue hormone replacement therapy prior to study enrollment due to the potential for inhibition of cytochrome p450 (cyp) enzymes that metabolize estrogens and progestins. * administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment. * have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to eltrombopag or excipients that contraindicate their participation.

Keywords: primacy immune thrombocytopenia adults eltrombopag long-term remission