1,3-丙酮二羧酸二乙酯化学性质熔点:125°C沸点:250°C(lit.)密度:1.113g/mLat25°C(lit.)折射率:n20/D1.440(lit.)闪点:187°F储存条件:Sealedindry,2-8°C溶解度:Chloroform(SlighChemicalbooktly),EthylAcetate(Slightly)酸度系数(pKa):9.66±0.46(Predicted)形态:Liquid颜色:Clearcolorlesstopaleyellow水溶解性:10g/L(20ºC)敏感性:MoistureSensitive
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Romiplostim is a recombinant fusion protein designed to mimic the activity of endogenous thrombopoietin (TPO), serving as a critical therapeutic agent for managing chronic immune thrombocytopenia (ITP). Structurally, it consists of a TPO receptor agonist domain fused to an Fc portion derived from human immunoglobulin G1. Its molecular formula is C285H453N79O87S6, and it is uniquely identified by the CAS number 402349-74-4. Unlike native TPO, Romiplostim is engineered to have a longer half-life in circulation while avoiding cross-reactivity with other cytokine receptors, ensuring high specificity for the c-Mpl receptor on megakaryocyte progenitor cells.
The primary clinical application of Romiplostim is the treatment of adult patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. By binding to the c-Mpl receptor on bone marrow megakaryocytes, it stimulates their proliferation and differentiation, thereby accelerating platelet production. This mechanism helps restore platelet counts to a safe range, significantly reducing the risk of bleeding events without the need for frequent blood transfusions. It is administered via subcutaneous injection once weekly, allowing for flexible dosing adjustments based on individual patient response and platelet count monitoring.
Safety profiles indicate that common adverse reactions include headache, nasopharyngitis, fatigue, and musculoskeletal pain. A rare but serious concern involves the potential increase in reticulin fibrosis in the bone marrow or the development of thrombotic complications, necessitating careful patient selection and regular hematological surveillance. While Romiplostim represents a significant advancement over traditional steroid therapies, offering a targeted approach to boost platelet production, its use requires strict adherence to dosing guidelines to avoid excessive thrombocytosis. As a non-glycosylated peptide, it offers manufacturing consistency and reduced immunogenicity compared to earlier generation agents. Ongoing research continues to explore its efficacy in other thrombocytopenic conditions, though its established role remains pivotal in refractory ITP management, providing a life-changing option for patients previously reliant on invasive procedures or long-term immunosuppression.