doi: 10.1111/nyas.14669
Examples included: Paroxysmal Nocturnal Haemoglobinuria (PNH) Immune Thrombocytopenia (ITP) Hemophagocytic Lymphohistiocytosis (HLH) Myelofibrosis Haemophilia von Willebrand disease, and other inherited bleeding disorders These studies examined treatment patterns, clinical outcomes, disease burden, healthcare utilization, and patient experiences outside controlled clinical trial environments
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However, most mechanistic evidence is derived from in vitro and animal studies, and the translation of these findings to human clinical outcomes remains limited
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