study on bone marrow morphology in adults receiving romiplostim for treatment of thrombocytopenia associated with immune thrombocytopenia purpura (itp)

Conditions: Thrombocytopenia; Idiopathic Thrombocytopenic Purpura

Summary: the purpose of this study is to evaluate changes in bone marrow morphology (structure) after long-term exposure to romiplostim.

participants diagnosed with itp according to the american society of hematology (ash) guidelines were sequentially enrolled into the following groups: * bone marrow biopsy at baseline and year 1 * bone marrow biopsy at baseline and year 2 * bone marrow biopsy at baseline and year 3. all participants received romiplostim for 3 years, unless withdrawn from the study early. participants returned for one visit for end of study (eos) procedures 4 weeks after romiplostim discontinuation, or, for participants who were withdrawn from the study due to the presence of collagen fibrosis, or had a change to grade 3 reticulin, at 12 weeks after discontinuation of romiplostim.

Eligibility criteria:
inclusion criteria: * diagnosis of itp according to american society of hematology (ash) guidelines * subject must have had a bone marrow biopsy within one year prior to planned first dose of romiplostim (with available bone marrow tissue block or unstained histological slides to send to a central laboratory for interpretation) or must consent to a pre-treatment bone marrow biopsy within 3 weeks prior to planned first dose of romiplostim. central laboratory interpretation is required prior to first dose of romiplostim * subject must agree to a scheduled bone marrow biopsy at year 1, year 2, or year 3 following romiplostim treatment and any unscheduled biopsies if clinically indicated * subject ≥18 years of age * baseline bone marrow reticulin grade of 0, 1, 2, or 3 according to the modified bauermeister grading scheme as assessed by central laboratory interpretation * platelet count \< 50 x 10\^9/l * must have received at least 1 prior itp therapy (examples of itp therapy include corticosteroids, intravenous immunoglobulin \[ivig\], splenectomy) * subject (or legally-acceptable representative) is willing and able to provide written informed consent exclusion criteria: * baseline bone marrow biopsy positive for collagen fibrosis * any known history of or currently active bone marrow stem cell disorder, hematological malignancy, myeloproliferative disorder, myelodysplastic syndrome * any current active malignancy * any prior exposure to cytostatic chemotherapy or radiotherapy for malignancy * subject has undergone pacemaker placement, cardiac ablation of arrhythmia, and/or any current treatment with vaughan williams class ia - ic and class iii agents (vaughan williams, 1970) * subject has participated in any study evaluating pegylated recombinant human megakaryocyte growth and development factor (peg-rhumgdf), recombinant human thrombopoietin (rhutpo), or thrombopoietin receptor agonists (ie romiplostim or eltrombopag) * subject has a known hypersensitivity to any recombinant e coli-derived product * subject is currently enrolled in or has not yet completed (at least 4 weeks since ending) other investigational device or drug trial(s) or subject is receiving other investigational agent(s) * other investigational procedures are excluded * subject of child-bearing potential is evidently pregnant (eg positive pregnancy test) or is breast feeding * subject is not using adequate contraceptive precautions * subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative and/or is unable to comply with study procedures.

Keywords: idiopathic thrombocytopenic purpura idiopathic thrombocytopenia purpura immune thrombocytopenic purpura immune thrombocytopenia itp