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Sökning: WFRF:(Weiler T) > (2020-2024) > Sustained Survival ...

Sustained Survival Benefit in Recurrent Medulloblastoma by a Metronomic Antiangiogenic Regimen: A Nonrandomized Controlled Trial.

Peyrl, Andreas (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.;Med Univ Vienna, Dept Pediat & Adolescent Med, Wahringer Gurtel 18-20, A-1090 Vienna, Austria.
Chocholous, Monika (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.
Sabel, Magnus, 1966 (författare)
Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics,Sahlgrens Univ Hosp, Queen Silvia Childrens Hosp, Childhood Canc Ctr, Gothenburg, Sweden.;Univ Gothenburg, Sahlgrenska Acad, Inst Clin Sci, Gothenburg, Sweden.
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Lassaletta, Alvaro (författare)
Hosp Infantil Univ Nino Jesus, Dept Pediat Neurooncol, Madrid, Spain.
Sterba, Jaroslav (författare)
Univ Hosp Brno, Pediat Oncol Dept, Brno, Czech Republic.
Leblond, Pierre (författare)
Oscar Lambret Comprehens Canc Ctr, Pediat Oncol Unit, Lille, France.;Inst Hematol & Doncol Pediat, Ctr Leon Berard, Lyon, France.
Nysom, Karsten (författare)
Rigshospitalet, Dept Paediat & Adolescent Med, Copenhagen, Denmark.
Torsvik, Ingrid (författare)
Haukeland Hosp, Dept Paediat & Adolescent Med, Bergen, Norway.
Chi, Susan N (författare)
Dana Farber Canc Inst, Dept Pediat Neurooncol, Boston, MA USA.
Perwein, Thomas (författare)
Med Univ Graz, Dept Pediat & Adolescent Med, Div Pediat Hemato Oncol, Graz, Austria.
Jones, Neil (författare)
Salzburger Univ klinikum, Kinderonkol, Salzburg, Austria.
Holm, Stefan (författare)
Karolinska Univ Hosp, Dept Pediat Hematol & Oncol, Stockholm, Sweden.
Nyman, Per (författare)
Linköpings universitet,Avdelningen för diagnostik och specialistmedicin,Medicinska fakulteten,Region Östergötland, H.K.H. Kronprinsessan Victorias barn- och ungdomssjukhus,Linkoping Univ Hosp, Dept Paediat, Linkoping, Sweden.
Mörse, Helena (författare)
Skane Univ Hosp, Pediat Canc Ctr, Lund, Sweden.
Öberg, Anders (författare)
Uppsala universitet,Barnonkologisk forskning - särskilt fokus på komplikationer,Uppsala Univ, Dept Pediat, Uppsala, Sweden.
Weiler-Wichtl, Liesa (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.
Leiss, Ulrike (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.
Haberler, Christine (författare)
Med Univ Vienna, Dept Neurol, Vienna, Austria.
Schmook, Maresa T (författare)
Med Univ Vienna, Dept Biomed Imaging & Image Guided Therapy, Div Neuroradiol & Musculoskeletal Radiol, Vienna, Austria.
Mayr, Lisa (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.
Dieckmann, Karin (författare)
Med Univ Vienna, Dept Radiooncol, Vienna, Austria.
Kool, Marcel (författare)
Hopp Childrens Canc Ctr KiTZ, Heidelberg, Germany.;German Canc Consortium DKTK, Div Pediat Neurooncol, German Canc Res Ctr DKFZ, Heidelberg, Germany.;Princess MaximaCtr Pediat Oncol, Utrecht, Netherlands.
Gojo, Johannes (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.
Azizi, Amedeo A (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.
André, Nicolas (författare)
Assistance Publ Hop Marseille, Dept Pediat Oncol, Marseille, France.;Aix Marseille Univ, Canc Res Ctr Marseille, Marseille, France.
Kieran, Mark (författare)
Dana Farber Canc Inst, Dept Pediat Neurooncol, Boston, MA USA.
Slavc, Irene (författare)
Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.
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Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria.;Med Univ Vienna, Dept Pediat & Adolescent Med, Wahringer Gurtel 18-20, A-1090 Vienna, Austria. Med Univ Vienna, Dept Pediat & Adolescent Med, Vienna, Austria.;Med Univ Vienna, Comprehens Ctr Pediat, Vienna, Austria. (creator_code:org_t)
AMER MEDICAL ASSOC, 2023
2023
Engelska.
Ingår i: JAMA oncology. - : AMER MEDICAL ASSOC. - 2374-2445 .- 2374-2437. ; 9:12, s. 1688-1695
  • Tidskriftsartikel (refereegranskat)
Abstract Ämnesord
Stäng  
  • 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.

Ämnesord

MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Pediatrik (hsv//swe)
MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Pediatrics (hsv//eng)
MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Cancer och onkologi (hsv//swe)
MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Cancer and Oncology (hsv//eng)
MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Hematologi (hsv//swe)
MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Hematology (hsv//eng)

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