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Clinical Trial
. 2021 Oct 20;39(30):3318-3327.
doi: 10.1200/JCO.20.02724. Epub 2021 Jul 20.

Comparison Between 5-Azacytidine Treatment and Allogeneic Stem-Cell Transplantation in Elderly Patients With Advanced MDS According to Donor Availability (VidazaAllo Study)

Nicolaus Kröger  1 , Katja Sockel  2 , Christine Wolschke  1 , Wolfgang Bethge  3 , Richard F Schlenk  4   5 , Dominik Wolf  6   7   8 , Michael Stadler  9 , Guido Kobbe  10 , Gerald Wulf  11 , Gesine Bug  12 , Kerstin Schäfer-Eckart  13 , Christof Scheid  14 , Florian Nolte  15 , Jan Krönke  16 , Matthias Stelljes  17 , Dietrich Beelen  18 , Marion Heinzelmann  1 , Detlef Haase  11 , Hannes Buchner  19 , Gabriele Bleckert  19 , Aristoteles Giagounidis  20 , Uwe Platzbecker  2   21
Affiliations
Clinical Trial

Comparison Between 5-Azacytidine Treatment and Allogeneic Stem-Cell Transplantation in Elderly Patients With Advanced MDS According to Donor Availability (VidazaAllo Study)

Nicolaus Kröger et al. J Clin Oncol. .

Abstract

Purpose: In contrast to 5-azacytidine (5-aza), allogeneic stem-cell transplantation (HSCT) represents a curative treatment strategy for patients with myelodysplastic syndromes (MDS), but therapy-related mortality (TRM) limits its broader use in elderly patients with MDS. The present prospective multicenter study compared HSCT following 5-aza pretreatment with continuous 5-aza treatment in patients with higher-risk MDS age 55-70 years.

Methods: One hundred ninety patients with a median age of 63 years were enrolled. Patients received 4-6 cycles of 5-aza followed by HLA-compatible HSCT after reduced-intensity conditioning or by continuous 5-aza if no donor was identified.

Results: Twenty-eight patients did not fulfill inclusion criteria (n = 20), died (n = 2) withdrew informed consent (n = 5), or were excluded for an unknown reason (n = 1). 5-aza induction started in 162 patients, but only 108 (67%) were eligible for subsequent allocation to HSCT (n = 81) or continuation of 5-aza (n = 27) because of disease progression (n = 26), death (n = 12), or other reasons (n = 16). Seven percent died during 5-aza before treatment allocation. The cumulative incidence of TRM after HSCT at 1 year was 19%. The event-free survival and overall survival after 5-aza pretreatment and treatment allocation at 3 years were 34% (95% CI, 22 to 47) and 50% (95% CI, 39 to 61) after allograft and 0% and 32% (95% CI, 14 to 52) after continuous 5-aza treatment (P < .0001 and P = .12), respectively. Fourteen patients progressing after continuous 5-aza received a salvage allograft from an alternative donor, and 43% were alive at last follow-up.

Conclusion: In older patients with MDS, reduced-intensity conditioning HSCT resulted in a significantly improved event-free survival in comparison with continuous 5-aza therapy. Bridging with 5-aza to HSCT before is associated with a considerable rate of dropouts because of progression, mortality, and adverse events.

Trial registration: ClinicalTrials.gov NCT01404741.

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Conflict of interest statement

Nicolaus KrögerHonoraria: Novartis, Celgene, Sanofi, Jazz Pharmaceuticals, Kite/Gilead, Riemser, AOP Orphan PharmaceuticalsConsulting or Advisory Role: Neovii, Sanofi, Jazz Pharmaceuticals, Novartis, Celgene, Riemser, Gilead SciencesSpeakers' Bureau: AOP Orphan PharmaceuticalsResearch Funding: Neovii, Novartis, Celgene, RiemserTravel, Accommodations, Expenses: Neovii, Novartis, Gilead Sciences, Jazz Pharmaceuticals, Sanofi, Celgene Katja SockelHonoraria: Bristol Myers Squibb/Celgene, Novartis, Alexion PharmaceuticalsConsulting or Advisory Role: Takeda Wolfgang BethgeConsulting or Advisory Role: Gilead Sciences, Novartis, Miltenyi BiotecSpeakers' Bureau: Miltenyi BiotecResearch Funding: Miltenyi BiotecTravel, Accommodations, Expenses: Gilead Sciences Richard F. SchlenkConsulting or Advisory Role: Daiichi Sankyo, PfizerSpeakers' Bureau: Pfizer, Daiichi Sankyo, NovartisResearch Funding: PharmaMar, AstraZeneca, Pfizer, Daiichi Sankyo, Boehringer Ingelheim, Roche Pharma AGTravel, Accommodations, Expenses: Daiichi Sankyo Guido KobbeHonoraria: Amgen, Jazz Pharmaceuticals, Neovii, Takeda, Medac, Biotest, Eurocept Pharmaceuticals, MSD, Roche, iQONE, Gilead Sciences, Celgene/Bristol Myers Squibb, AbbVie, Novartis, PfizerConsulting or Advisory Role: Celgene/Bristol Myers Squibb, Amgen, Pfizer, Jazz Pharmaceuticals, Neovii, Takeda, Medac, Biotest, Eurocept Pharmaceuticals, MSD, Roche, iQONE, Novartis, Gilead Sciences, AbbVieResearch Funding: Celgene/Bristol Myers Squibb, AmgenTravel, Accommodations, Expenses: Pfizer, AbbVie, Amgen, MSD, Novartis Gerald WulfHonoraria: Hematology TodayConsulting or Advisory Role: Gilead Sciences, Novartis, Takeda, Clinigen GroupSpeakers' Bureau: Takeda Gesine BugHonoraria: Jazz Pharmaceuticals, CelgeneConsulting or Advisory Role: Hexal, Novartis, Pfizer, Eurocept, Gilead Sciences, CelgeneResearch Funding: NovartisTravel, Accommodations, Expenses: Gilead Sciences, Sanofi, Celgene, Neovii Christof ScheidHonoraria: Amgen, Bristol Myers Squibb/Celgene, Janssen Oncology, Novartis, Pfizer, TakedaConsulting or Advisory Role: Amgen, Roche, Janssen Oncology, Bristol Myers Squibb/CelgeneResearch Funding: Janssen Oncology, TakedaTravel, Accommodations, Expenses: Bristol Myers Squibb/Celgene, Janssen Oncology, Amgen Jan KrönkeConsulting or Advisory Role: Takeda, Janssen, Sanofi, Bristol Myers Squibb/Celgene Matthias StelljesConsulting or Advisory Role: Pfizer, Jazz Pharmaceuticals, Gilead Sciences, MSD, AmgenSpeakers' Bureau: Pfizer, Medac, MSD, IncyteResearch Funding: PfizerTravel, Accommodations, Expenses: Medac, Neovii Dietrich BeelenHonoraria: MedacConsulting or Advisory Role: Medac Detlef HaaseHonoraria: Novartis, Jazz Pharmaceuticals, Takeda, Celgene/Bristol Myers SquibbTravel, Accommodations, Expenses: Celgene/Bristol Myers Squibb Hannes BuchnerEmployment: Staburo GmbHLeadership: Staburo GmbHOther Relationship: Staburo GmbH Aristoteles GiagounidisStock and Other Ownership Interests: Novartis, RocheHonoraria: Celgene, Amgen, NovartisConsulting or Advisory Role: Celgene Uwe PlatzbeckerHonoraria: Celgene/JazzConsulting or Advisory Role: Celgene/JazzResearch Funding: Amgen, Janssen, Novartis, BerGenBio, CelgenePatents, Royalties, Other Intellectual Property: Part of a patent for a TFR-2 antibody (Rauner et al. Nature Metabolics 2019)Travel, Accommodations, Expenses: CelgeneNo other potential conflicts of interest were reported.

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