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@ARTICLE{Hegenbart:128052,
      author       = {U. Hegenbart and T. Bochtler$^*$ and A. Benner$^*$ and N.
                      Becker$^*$ and C. Kimmich and A. V. Kristen and J. Beimler
                      and E. Hund and M. Zorn and A. Freiberger and M. Gawlik and
                      H. Goldschmidt$^*$ and D. Hose and A. Jauch and A. D. Ho and
                      S. O. Schönland},
      title        = {{L}enalidomide/melphalan/dexamethasone in newly diagnosed
                      patients with immunoglobulin light chain amyloidosis:
                      results of a prospective phase 2 study with long-term follow
                      up.},
      journal      = {Haematologica},
      volume       = {102},
      number       = {8},
      issn         = {1592-8721},
      address      = {Pavia},
      publisher    = {Ferrata Storti Foundation},
      reportid     = {DKFZ-2017-04074},
      pages        = {1424 - 1431},
      year         = {2017},
      abstract     = {Chemotherapy in light chain amyloidosis aims to normalize
                      the involved free light chain in serum, which leads to an
                      improvement, or at least stabilization of organ function in
                      most responding patients. We performed a prospective single
                      center phase 2 trial with 50 untreated patients not eligible
                      for high-dose treatment. The treatment schedule comprised 6
                      cycles of oral lenalidomide, melphalan and dexamethasone
                      every 4 weeks. After 6 months, complete remission was
                      achieved in 9 patients $(18\%),$ very good partial remission
                      in 16 $(32\%)$ and partial response in 9 $(18\%).$ Overall,
                      organ response was observed in 24 patients $(48\%).$
                      Hematologic and cardiac toxicities were predominant adverse
                      events. Mortality at 3 months was low at $4\%$ (n=2) despite
                      the inclusion of $36\%$ of patients (n=18) with cardiac
                      stage Mayo 3. After a median follow-up of 50 months, median
                      overall and event-free survival were 67.5 months and 25.1
                      months, respectively. We conclude that the treatment of
                      lenalidomide, melphalan and dexamethasone is very effective
                      in achieving a hematologic remission, organ response and,
                      consecutively, a long survival in transplant ineligible
                      patients with light chain amyloidosis. However, as toxicity
                      and tolerability are the major problems of a 3-drug regimen,
                      a strict surveillance program is necessary and sufficient to
                      avoid severe toxicities. clinicaltrials.gov Identifier:
                      00883623 (Eudract2008-001405-41).},
      cin          = {G330 / C060 / V964},
      ddc          = {610},
      cid          = {I:(DE-He78)G330-20160331 / I:(DE-He78)C060-20160331 /
                      I:(DE-He78)V964-20160331},
      pnm          = {319H - Addenda (POF3-319H)},
      pid          = {G:(DE-HGF)POF3-319H},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:28522573},
      pmc          = {pmc:PMC5541875},
      doi          = {10.3324/haematol.2016.163246},
      url          = {https://inrepo02.dkfz.de/record/128052},
}