Bone Marrow Transplantation

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ISSN / EISSN : 0268-3369 / 1476-5365
Current Publisher: Springer Science and Business Media LLC (10.1038)
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Total articles ≅ 9,798
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Yngvar Fløisand, Mark A. Schroeder, Patrice Chevallier, Dominik Selleslag, Steven Devine, Anne S. Renteria, , , Chunlin Chen, Andrejus Parfionovas, et al.
Bone Marrow Transplantation pp 1-12; doi:10.1038/s41409-021-01356-0

Abstract:
Steroid-refractory (SR) acute graft-versus-host disease (aGvHD) remains a significant complication after allogeneic hematopoietic cell transplantation. Systemic corticosteroids are first-line therapy for aGvHD, but apart from ruxolitinib, there are no approved treatments for SR aGvHD. Vedolizumab is approved for treatment of ulcerative colitis and Crohn’s disease, and may be effective for treatment of SR intestinal aGvHD. We conducted a phase 2a trial (NCT02993783) to evaluate the clinical efficacy, tolerability, and safety of vedolizumab 300 and 600 mg for SR intestinal aGvHD. This study was terminated before full enrollment was completed because early results failed to demonstrate positive proof-of-concept in efficacy. Before termination, 17 participants had enrolled and an early response in intestinal aGvHD was observed in 11 and eight participants at days 15 and 28, respectively. All adverse events observed were consistent with those expected in a population with SR intestinal aGvHD. Overall, vedolizumab did not meet the primary efficacy endpoint (overall response at day 28), likely owing to premature study drug discontinuation, lack of efficacy, and the competing risks inherent with a population with advanced SR intestinal aGvHD. Nevertheless, this study provides valuable insights into the considerations needed when conducting studies in patients with SR intestinal aGvHD.
, Irene Defrancesco, , Francesco Barbato, Paolo Lopedote, Michele Dicataldo, Mariarosaria Sessa, Elisa Dan, Barbara Sinigaglia, Luca Zazzeroni, et al.
Bone Marrow Transplantation pp 1-4; doi:10.1038/s41409-021-01359-x

Stephanie Mac, Dat Ngo, Dongyun Yang, , , Shukaib Arslan, Sanjeet Dadwal, Amandeep Salhotra, Thai Cao, Nicole Karras, et al.
Bone Marrow Transplantation pp 1-7; doi:10.1038/s41409-021-01364-0

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, Eri Nishikawa, Atsunari Tsuchisaka, Takashi Hashimoto, Yusuke Okuno, Motoharu Hamada, Daisuke Ichikawa, , , Nobuhiro Nishio, et al.
Bone Marrow Transplantation pp 1-4; doi:10.1038/s41409-021-01335-5

Nassim Ait Abdallah, Mianbo Wang, Pauline Lansiaux, Mathieu Puyade, Sabine Berthier, Louis Terriou, Catney Charles, , Marie Hudson,
Bone Marrow Transplantation pp 1-9; doi:10.1038/s41409-021-01355-1

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Ram Vasudevan Nampoothiri, Lina Ho, Cassandra McEwan, Ivan Pasic, Wilson Lam, , Fotios V. Michelis, Santhosh Thyagu, Dennis (Dong Hwan) Kim, Armin Gerbitz, et al.
Bone Marrow Transplantation pp 1-6; doi:10.1038/s41409-021-01362-2

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, Myriam Labopin, , Nicolaus Kröger, Jan Cornelissen, Tobias Gedde-Dahl, Gwendolyn Van Gorkom, Jürgen Finke, Montserrat Rovira, Nicolaas Schaap, et al.
Bone Marrow Transplantation pp 1-9; doi:10.1038/s41409-021-01322-w

Abstract:
Acute myeloid leukemia with runt-related transcription factor 1 gene mutation (RUNX1+ AML) is associated with inferior response rates and outcome after conventional chemotherapy. We performed a retrospective, registry-based analysis to elucidate the prognostic value of RUNX1 mutation after allogeneic stem cell transplantation (alloSCT). All consecutive adults undergoing alloSCT for AML in first complete remission (CR1) between 2013 and 2019 with complete information on conventional cytogenetics and RUNX1 mutational status were included. Endpoints of interest were cumulative relapse incidence, non-relapse mortality, overall and leukemia-free survival (OS/LFS), and GvHD-free/relapse-free survival. A total of 674 patients (183 RUNX1+, 491 RUNX1−) were identified, with >85% presenting as de novo AML. Median follow-up was 16.4 (RUNX1+) and 21.9 (RUNX1−) months. Survival rates showed no difference between RUNX1+ and RUNX1− patients either in univariate or multivariate analysis (2-year OS: 67.7 vs. 66.1%, p = 0.7; 2-year LFS: 61.1 vs. 60.8%, p = 0.62). Multivariate analysis identified age, donor type and poor cytogenetics as risk factors for inferior outcome. Among patients with RUNX+ AML, older age, reduced intensity conditioning and minimal residual disease at alloSCT predicted inferior outcome. Our data provide evidence that the negative influence of RUNX1 mutations in patients with AML can be overcome by transplantation in CR1.
, Spanish Group for Hematopoietic Stem cell Transplantation (GETH), Maria Jesús Pascual, Mi Kwon, Ariadna Pérez, Rocio Parody, Christelle Ferra, , Beatriz Herruzo, Nieves Dorado, et al.
Bone Marrow Transplantation; doi:10.1038/s41409-021-01328-4

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