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@ARTICLE{Vernava:212442,
      author       = {I. Vernava and C. A. Schmitt$^*$},
      title        = {{D}aratumumab as a novel treatment option in refractory
                      {ITP}.},
      journal      = {Blood cells, molecules $\&$ diseases},
      volume       = {99},
      issn         = {1079-9796},
      address      = {Orlando, Fla.},
      publisher    = {Academic Press},
      reportid     = {DKFZ-2023-00161},
      pages        = {102724},
      year         = {2023},
      abstract     = {Primary immune thrombocytopenia (ITP) in adult patients
                      typically presents as a repeatedly relapsing disease in need
                      of multiple lines of therapy. Here we report the clinical
                      courses of two patients, an 82-year-old female and a
                      54-year-old male, with primary ITP after multiple relapses
                      and exhausted standard therapies, which we treated with the
                      myeloma-licensed anti-CD38 monoclonal antibody daratumumab
                      in an off-label setting. Daratumumab is known to target
                      preferentially plasmablasts, short-lived plasma cells and
                      long-lived plasma cells, with the latter being the major
                      source of antiplatelet autoantibodies. Noteworthy,
                      rituximab, a CD20 antibody, targets earlier steps in B-cell
                      ontogenesis, thereby indirectly decreasing plasmablasts and
                      short-lived plasma cells, but to a lesser extent long-lived
                      plasma cells, which tend to persist after rituximab
                      treatment. Several single-patient reports and case series
                      have demonstrated successful treatment with daratumumab in
                      ITP, autoimmune thrombocytopenia in Evans syndrome as well
                      as other cytopenias or pure red cell aplasia after
                      allogeneic stem cell transplantation or in congenital
                      diseases, systemic lupus erythematodes and cold agglutinin
                      disease. Our first patient with isolated primary ITP rapidly
                      and lastingly responded to daratumumab plus tapered
                      steroids, with platelet counts above 50 × 109/L within
                      weeks and subsequently even stably within the normal range.
                      Despite no objective response observed in the second
                      patient, a lasting clinical stabilization was achieved. As
                      the underlying mode of action, we hypothesize here
                      daratumumab to effectively target long-lived plasma cells as
                      the source of ITP-mediating autoantibodies, and suggest
                      broader clinical evaluation of daratumumab in this potential
                      indication.},
      keywords     = {CD38 (Other) / Daratumumab (Other) / ITP (Other) / Immune
                      thrombocytopenia (Other) / Long-lived plasma cells (Other)},
      cin          = {BE01},
      ddc          = {610},
      cid          = {I:(DE-He78)BE01-20160331},
      pnm          = {899 - ohne Topic (POF4-899)},
      pid          = {G:(DE-HGF)POF4-899},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:36669360},
      doi          = {10.1016/j.bcmd.2023.102724},
      url          = {https://inrepo02.dkfz.de/record/212442},
}