Immune Thrombocytopenia — Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia
Citation(s)
Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3. Erratum in: Blood. 2009 May 7;113(19):4822.
Cooper N, Bussel J The pathogenesis of immune thrombocytopaenic purpura. Br J Haematol. 2006 May;133(4):364-74. Review.
Wong RSM, Saleh MN, Khelif A, Salama A, Portella MSO, Burgess P, Bussel JB Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017 Dec 7;130(23):2527-2536. doi: 10.1182/blood-2017-04-748707. Epub 2017 Oct 17. Erratum in: Blood. 2018 Feb 8;131(6):709.
Phase II Trial of Romiplostim With Danazol in Patients With Eltrombopag-resistant Immune Thrombocytopenia
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.
