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@ARTICLE{Peyrl:285006,
      author       = {A. Peyrl and M. Chocholous and M. Sabel and A. Lassaletta
                      and J. Sterba and P. Leblond and K. Nysom and I. Torsvik and
                      S. N. Chi and T. Perwein and N. Jones and S. Holm and P.
                      Nyman and H. Mörse and A. Öberg and L. Weiler-Wichtl and
                      U. Leiss and C. Haberler and M. T. Schmook and L. Mayr and
                      K. Dieckmann and M. Kool$^*$ and J. Gojo and A. A. Azizi and
                      N. André and M. Kieran and I. Slavc},
      title        = {{S}ustained {S}urvival {B}enefit in {R}ecurrent
                      {M}edulloblastoma by a {M}etronomic {A}ntiangiogenic
                      {R}egimen: {A} {N}onrandomized {C}ontrolled {T}rial.},
      journal      = {JAMA oncology},
      volume       = {9},
      number       = {12},
      issn         = {2374-2437},
      address      = {Chicago, Ill.},
      publisher    = {American Medical Association},
      reportid     = {DKFZ-2023-02179},
      pages        = {1688-1695},
      year         = {2023},
      note         = {2023 Dec 1;9(12):1688-1695},
      abstract     = {Medulloblastoma recurrence in patients who have previously
                      received irradiation has a dismal prognosis and lacks a
                      standard salvage regimen.To evaluate the response rate of
                      pediatric patients with medulloblastoma recurrence using an
                      antiangiogenic metronomic combinatorial approach
                      (Medulloblastoma European Multitarget Metronomic
                      Anti-Angiogenic Trial [MEMMAT]).This phase 2,
                      investigator-initiated, multicenter nonrandomized controlled
                      trial assessed 40 patients with relapsed or refractory
                      medulloblastoma without a ventriculoperitoneal shunt who
                      were younger than 20 years at original diagnosis. Patients
                      were enrolled between April 1, 2014, and March 31,
                      2021.Treatment consisted of daily oral thalidomide,
                      fenofibrate, celecoxib, and alternating 21-day cycles of
                      low-dose (metronomic) oral etoposide and cyclophosphamide,
                      supplemented by intravenous bevacizumab and intraventricular
                      therapy consisting of alternating etoposide and
                      cytarabine.The primary end point was response after 6 months
                      of antiangiogenic metronomic therapy. Secondary end points
                      included progression-free survival (PFS), overall survival
                      (OS), and quality of life. Adverse events were monitored to
                      assess safety.Of the 40 patients (median [range] age at
                      treatment start, 10 [4-17] years; 25 $[62.5\%]$ male)
                      prospectively enrolled, 23 $(57.5\%)$ achieved disease
                      control after 6 months of treatment, with a response
                      detected in 18 patients $(45.0\%).$ Median OS was 25.5
                      months (range, 10.9-40.0 months), and median PFS was 8.5
                      months (range, 1.7-15.4 months). Mean (SD) PFS at both 3 and
                      5 years was $24.6\%$ $(7.9\%),$ while mean (SD) OS at 3 and
                      5 years was $43.6\%$ $(8.5\%)$ and $22.6\%$ $(8.8\%),$
                      respectively. No significant differences in PFS or OS were
                      evident based on molecular subgroup analysis or the number
                      of prior recurrences. In patients demonstrating a response,
                      mean (SD) overall 5-year PFS was $49.7\%$ $(14.3\%),$ and
                      for patients who remained progression free for the first 12
                      months of treatment, mean (SD) 5-year PFS was $66.7\%$
                      $(16.1\%).$ Treatment was generally well tolerated. Grade 3
                      to 4 treatment-related adverse events included
                      myelosuppression, infections, seizures, and headaches. One
                      heavily pretreated patient with a third recurrence died of
                      secondary acute myeloid leukemia.This feasible and
                      well-tolerated MEMMAT combination regimen demonstrated
                      promising activity in patients with previously irradiated
                      recurrent medulloblastoma. Given these results, this
                      predominantly oral, well-tolerated, and outpatient treatment
                      warrants further evaluation.ClinicalTrials.gov Identifier:
                      NCT01356290.},
      cin          = {B062 / HD01},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:37883081},
      doi          = {10.1001/jamaoncol.2023.4437},
      url          = {https://inrepo02.dkfz.de/record/285006},
}