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Issue Archive

Volume 5, Issue 5
March 9 2021
Issue Cover

Table of Contents

DRUG ADVANCES

Professional illustration by Patrick Lane, ScEYEnce Studios.

Development of luspatercept to treat ineffective erythropoiesis
Clinical Trials & Observations
Anne Sophie Kubasch,Pierre Fenaux,Uwe Platzbecker
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EXCEPTIONAL CASE REPORT

Single-cell DNA sequencing reveals complex mechanisms of resistance to quizartinib
Clinical Trials & Observations
Cheryl A. C. Peretz,Lisa H. F. McGary,Tanya Kumar,Hunter Jackson,Jose Jacob,Robert Durruthy-Durruthy,Mark J. Levis,Alexander Perl,Benjamin J. Huang,Catherine C. Smith
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Supplemental data

STIMULUS REPORTS

Microbial dysbiosis is associated with aggressive histology and adverse clinical outcome in B-cell non-Hodgkin lymphoma
Clinical Trials & Observations
Catherine S. Diefenbach,Brandilyn A. Peters,Huilin Li,Bruce Raphael,Tibor Moskovits,Kenneth Hymes,Jonas Schluter,J. Chen,N. Nora Bennani,Thomas E. Witzig,Jiyoung Ahn
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Supplemental data
Disruption of CSF-1R signaling inhibits growth of AML with inv(16)
Alexander Simonis,Norman F. Russkamp,Jan Mueller,C. Matthias Wilk,Mattheus H. E. Wildschut,Renier Myburgh,Nicole Wildner-Verhey van Wijk,Rouven Mueller,Stefan Balabanov,Peter J. M. Valk,Alexandre P. A. Theocharides,Markus G. Manz
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Supplemental data
Whole-blood phenotyping to assess alloimmunization status in transfused sickle cell disease patients
Marie Tamagne,Sadaf Pakdaman,Pablo Bartolucci,Anoosha Habibi,Frédéric Galactéros,France Pirenne,Benoît Vingert
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Supplemental data
The SARS-CoV-2 receptor-binding domain preferentially recognizes blood group A
Shang-Chuen Wu,Connie M. Arthur,Jianmei Wang,Hans Verkerke,Cassandra D. Josephson,Daniel Kalman,John D. Roback,Richard D. Cummings,Sean R. Stowell
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Supplemental data
HLA-haploidentical TCRαβ+/CD19+-depleted stem cell transplantation in children and young adults with Fanconi anemia
Clinical Trials & Observations
Luisa Strocchio,Daria Pagliara,Mattia Algeri,Giuseppina Li Pira,Francesca Rossi,Valentina Bertaina,Giovanna Leone,Rita Maria Pinto,Marco Andreani,Emanuele Agolini,Katia Girardi,Stefania Gaspari,Lavinia Grapulin,Francesca del Bufalo,Antonio Novelli,Pietro Merli,Franco Locatelli
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REVIEW ARTICLE

Platelets as drivers of ischemia/reperfusion injury after stroke
Noor F. Shaik,Raymond F. Regan,Ulhas P. Naik
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CLINICAL TRIALS AND OBSERVATIONS

A phase 2 trial of GVHD prophylaxis with PTCy, sirolimus, and MMF after peripheral blood haploidentical transplantation
Clinical Trials & Observations
Nelli Bejanyan,Joseph A. Pidala,Xuefeng Wang,Ram Thapa,Taiga Nishihori,Hany Elmariah,Aleksandr Lazaryan,Farhad Khimani,Marco L. Davila,Asmita Mishra,Rawan Faramand,Michael D. Jain,Leonel Ochoa,Lia Elena Perez,Hien Liu,Melissa Alsina,Mohamed A. Kharfan-Dabaja,Hugo Fernandez,Michael L. Nieder,Frederick L. Locke,Claudio Anasetti,Ernesto Ayala
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Outcomes and prognostic factors in adolescents and young adults with ALL treated with a modified BFM-90 protocol
Clinical Trials & Observations
Akhil Rajendra,Hasmukh Jain,V. N. Avinash Bonda,Lingaraj Nayak,Prashant Tembhare,Dhanlaxmi Shetty,Jayashree Thorat,Hemani Jain,Papagudi Ganesan Subramanian,Nikhil Patkar,Gaurav Chatterjee,Navin Khattry,Anant Gokarn,Sachin Punatar,Smruti Mokal,Bhausaheb Bagal,Manju Sengar
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Supplemental data
Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial
Clinical Trials & Observations
Yaqiong Tang,Jia Chen,Qifa Liu,Tiantian Chu,Tingting Pan,Jianying Liang,Xue Feng He,Feng Chen,Ting Yang,Xiao Ma,Xiaojin Wu,Shaoyan Hu,Xingyu Cao,Xiaohui Hu,Jiong Hu,Yuejun Liu,Jiaqian Qi,Yueping Shen,Changgeng Ruan,Yue Han,Depei Wu
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Supplemental data
End-of-treatment PET/CT predicts PFS and OS in DLBCL after first-line treatment: results from GOYA
Clinical Trials & Observations
Lale Kostakoglu,Maurizio Martelli,Laurie H. Sehn,David Belada,Angelo-Michele Carella,Neil Chua,Eva Gonzalez-Barca,Xiaonan Hong,Antonio Pinto,Yuankai Shi,Yoichi Tatsumi,Andrea Knapp,Federico Mattiello,Tina Nielsen,Deniz Sahin,Gila Sellam,Mikkel Z. Oestergaard,Umberto Vitolo,Marek Trněný
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Supplemental data
Long-term outcomes of R-CEOP show curative potential in patients with DLBCL and a contraindication to anthracyclines
Clinical Trials & Observations
Alden A. Moccia,Kimberly Schaff,Ciara Freeman,Paul J. Hoskins,Richard J. Klasa,Kerry J. Savage,Tamara N. Shenkier,Randy D. Gascoyne,Joseph M. Connors,Laurie H. Sehn
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Supplemental data
Heterogeneous NLRP3 inflammasome signature in circulating myeloid cells as a biomarker of COVID-19 severity
Clinical Trials & Observations
Johan Courjon,Océane Dufies,Alexandre Robert,Laurent Bailly,Cédric Torre,David Chirio,Julie Contenti,Sébastien Vitale,Céline Loubatier,Anne Doye,Christelle Pomares-Estran,Géraldine Gonfrier,Romain Lotte,Patrick Munro,Orane Visvikis,Jean Dellamonica,Valérie Giordanengo,Michel Carles,Laurent Yvan-Charvet,Stoyan Ivanov,Patrick Auberger,Arnaud Jacquel,Laurent Boyer
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Supplemental data
Tandem high-dose influenza vaccination is associated with more durable serologic immunity in patients with plasma cell dyscrasias
Clinical Trials & Observations
Andrew R. Branagan,Eamon Duffy,Geliang Gan,Fangyong Li,Connor Foster,Rakesh Verma,Lin Zhang,Terri L. Parker,Stuart Seropian,Dennis L. Cooper,Debra Brandt,Jeremy Kortmansky,Davit Witt,Thomas M. Ferencz,Kavita M. Dhodapkar,Madhav V. Dhodapkar
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Supplemental data

GENE THERAPY

Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
Giulia Pavani,Anna Fabiano,Marine Laurent,Fatima Amor,Erika Cantelli,Anne Chalumeau,Giulia Maule,Alexandra Tachtsidi,Jean-Paul Concordet,Anna Cereseto,Fulvio Mavilio,Giuliana Ferrari,Annarita Miccio,Mario Amendola
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Supplemental data
Single-dose MGTA-145/plerixafor leads to efficient mobilization and in vivo transduction of HSCs with thalassemia correction in mice
Chang Li,Kevin A. Goncalves,Tamás Raskó,Amit Pande,Sucheol Gil,Zhinan Liu,Zsuzsanna Izsvák,Thalia Papayannopoulou,John C. Davis, Jr,Hans-Peter Kiem,André Lieber
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Supplemental data

HEALTH SERVICES AND OUTCOMES

Comorbidities and malignancies negatively affect survival in myelodysplastic syndromes: a population-based study
Clinical Trials & Observations
Johanne Rozema,Mels Hoogendoorn,Robby Kibbelaar,Eva van den Berg,Nic Veeger,Eric van Roon,on behalf of the HemoBase Population Registry Consortium
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Supplemental data

HEMATOPOIESIS AND STEM CELLS

A CRISPR RNA-binding protein screen reveals regulators of RUNX1 isoform generation
Amanda G. Davis,Jaclyn M. Einstein,Dinghai Zheng,Nathan D. Jayne,Xiang-Dong Fu,Bin Tian,Gene W. Yeo,Dong-Er Zhang
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Supplemental data

IMMUNOBIOLOGY AND IMMUNOTHERAPY

A BCMAxCD3 bispecific T cell–engaging antibody demonstrates robust antitumor efficacy similar to that of anti-BCMA CAR T cells
David J. DiLillo,Kara Olson,Katja Mohrs,Thomas Craig Meagher,Kevin Bray,Olga Sineshchekova,Thomas Startz,Jessica Kuhnert,Marc W. Retter,Stephen Godin,Prachi Sharma,Frank Delfino,John Lin,Eric Smith,Gavin Thurston,Jessica R. Kirshner
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Supplemental data

LYMPHOID NEOPLASIA

Genome-wide interference of ZNF423 with B-lineage transcriptional circuitries in acute lymphoblastic leukemia
Pablo Iglesias,Ann-Christin Puller,Marcos Seoane,Michael Spohn,Sabine Raasch,Marianne Klokow,Jürgen Müller,Lia Burkhardt,Daniela Indenbirken,Martin A. Horstmann
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Supplemental data
Clinical manifestations of, diagnostic approach to, and treatment of neurolymphomatosis in the rituximab era
Clinical Trials & Observations
Arushi Khurana,Mattia Novo,Grzegorz S. Nowakowski,Kay M. Ristow,Robert J. Spinner,Christopher H. Hunt,Rebecca L. King,Daniel H. Lachance,Thomas M. Habermann,Ivana N. Micallef,Patrick B. Johnston
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Supplemental data
Impact of event-free survival status after stem cell transplantation on subsequent survival of patients with lymphoma
Ayumi Fujimoto,Tatsuhiko Anzai,Takahiro Fukuda,Naoyuki Uchida,Takanori Ohta,Takehiko Mori,Masashi Sawa,Satoshi Yoshioka,Toshihiro Miyamoto,Hitoji Uchiyama,Yuta Katayama,Ken-ichi Matsuoka,Souichi Shiratori,Hideyuki Nakazawa,Junya Kanda,Tatsuo Ichinohe,Yoshiko Atsuta,Naoto Fujita,Eisei Kondo,Ritsuro Suzuki
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Supplemental data

MYELOID NEOPLASIA

Molecular response to imatinib in patients with chronic myeloid leukemia in Tanzania
Clinical Trials & Observations Global Advances
Ahlam Nasser,Ally Hussein,Clara Chamba,Mbonea Yonazi,Rosemary Mushi,Anna Schuh,Lucio Luzzatto
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Supplemental data
Genomic analysis of primary and secondary myelofibrosis redefines the prognostic impact of ASXL1 mutations: a FIM study
Clinical Trials & Observations
Damien Luque Paz,Jérémie Riou,Emmanuelle Verger,Bruno Cassinat,Aurélie Chauveau,Jean-Christophe Ianotto,Brigitte Dupriez,Françoise Boyer,Maxime Renard,Olivier Mansier,Anne Murati,Jérôme Rey,Gabriel Etienne,Véronique Mansat-De Mas,Suzanne Tavitian,Olivier Nibourel,Stéphane Girault,Yannick Le Bris,François Girodon,Dana Ranta,Jean-Claude Chomel,Pascale Cony-Makhoul,Pierre Sujobert,Margot Robles,Raouf Ben Abdelali,Olivier Kosmider,Laurane Cottin,Lydia Roy,Ivan Sloma,Fabienne Vacheret,Mathieu Wemeau,Pascal Mossuz,Borhane Slama,Vincent Cussac,Guillaume Denis,Anouk Walter-Petrich,Barbara Burroni,Nathalie Jézéquel,Stéphane Giraudier,Eric Lippert,Gérard Socié,Jean-Jacques Kiladjian,Valérie Ugo,on behalf of the French Intergroup of Myeloproliferative Neoplasms
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Supplemental data
Gene expression profile correlates with molecular and clinical features in patients with myelofibrosis
Clinical Trials & Observations
Sebastiano Rontauroli,Sara Castellano,Paola Guglielmelli,Roberta Zini,Elisa Bianchi,Elena Genovese,Chiara Carretta,Sandra Parenti,Sebastian Fantini,Selene Mallia,Lara Tavernari,Stefano Sartini,Margherita Mirabile,Carmela Mannarelli,Francesca Gesullo,Annalisa Pacilli,Daniela Pietra,Elisa Rumi,Silvia Salmoiraghi,Barbara Mora,Laura Villani,Andrea Grilli,Vittorio Rosti,Giovanni Barosi,Francesco Passamonti,Alessandro Rambaldi,Luca Malcovati,Mario Cazzola,Silvio Bicciato,Enrico Tagliafico,Alessandro M. Vannucchi,Rossella Manfredini,on behalf of the Mynerva (MYeloid NEoplasms Research Venture AIRC) investigators
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Supplemental data
Gene expression signature predicts relapse in adult patients with cytogenetically normal acute myeloid leukemia
Clinical Trials & Observations
Christopher J. Walker,Krzysztof Mrózek,Hatice Gulcin Ozer,Deedra Nicolet,Jessica Kohlschmidt,Dimitrios Papaioannou,Luke K. Genutis,Marius Bill,Bayard L. Powell,Geoffrey L. Uy,Jonathan E. Kolitz,Andrew J. Carroll,Richard M. Stone,Ramiro Garzon,John C. Byrd,Ann-Kathrin Eisfeld,Albert de la Chapelle,Clara D. Bloomfield
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Supplemental data
Deregulated expression of circular RNAs in acute myeloid leukemia
Susanne Lux,Tamara J. Blätte,Bernhard Gillissen,Antje Richter,Sibylle Cocciardi,Sabrina Skambraks,Klaus Schwarz,Hubert Schrezenmeier,Hartmut Döhner,Konstanze Döhner,Anna Dolnik,Lars Bullinger
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Supplemental data
Transcriptomic and genomic heterogeneity in blastic plasmacytoid dendritic cell neoplasms: from ontogeny to oncogenesis
Florian Renosi,Anne Roggy,Ambre Giguelay,Lou Soret,Pierre-Julien Viailly,Meyling Cheok,Sabeha Biichle,Fanny Angelot-Delettre,Vahid Asnafi,Elizabeth Macintyre,Sandrine Geffroy,Mary Callanan,Tony Petrella,Eric Deconinck,Etienne Daguindau,Véronique Harrivel,Sabrina Bouyer,Véronique Salaun,Pascale Saussoy,Jean Feuillard,Pascal Fuseau,Philippe Saas,Olivier Adotévi,Fabrice Jardin,Christophe Ferrand,Claude Preudhomme,Jacques Colinge,Christophe Roumier,Francine Garnache-Ottou
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Supplemental data
Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed/refractory AML
Clinical Trials & Observations
Maximilian Stahl,Kamal Menghrajani,Andriy Derkach,Alexander Chan,Wenbin Xiao,Jacob Glass,Amber C. King,Anthony F. Daniyan,Christopher Famulare,Bernadette M. Cuello,Troy Z. Horvat,Omar Abdel-Wahab,Ross L. Levine,Aaron D. Viny,Eytan M. Stein,Sheng F. Cai,Mikhail Roshal,Martin S. Tallman,Aaron D. Goldberg
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Supplemental data

PHAGOCYTES, GRANULOCYTES, AND MYELOPOIESIS

A neutrophil activation signature predicts critical illness and mortality in COVID-19
Clinical Trials & Observations
Matthew L. Meizlish,Alexander B. Pine,Jason D. Bishai,George Goshua,Emily R. Nadelmann,Michael Simonov,C-Hong Chang,Hanming Zhang,Marcus Shallow,Parveen Bahel,Kent Owusu,Yu Yamamoto,Tanima Arora,Deepak S. Atri,Amisha Patel,Rana Gbyli,Jennifer Kwan,Christine H. Won,Charles Dela Cruz,Christina Price,Jonathan Koff,Brett A. King,Henry M. Rinder,F. Perry Wilson,John Hwa,Stephanie Halene,William Damsky,David van Dijk,Alfred I. Lee,Hyung J. Chun
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Supplemental data
Proteomic and bioinformatic profiling of neutrophils in CLL reveals functional defects that predispose to bacterial infections
Nirojah Subramaniam,Jenny Bottek,Stephanie Thiebes,Kristina Zec,Matthias Kudla,Camille Soun,Elena de Dios Panal,Julia K. Lill,Aaron Pfennig,Ralf Herrmann,Kirsten Bruderek,Sven Rahmann,Sven Brandau,Patricia Johansson,Hans Christian Reinhardt,Jan Dürig,Martina Seiffert,Thilo Bracht,Barbara Sitek,Daniel Robert Engel
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Supplemental data

RED CELLS, IRON, AND ERYTHROPOIESIS

MetAP2 inhibition modifies hemoglobin S to delay polymerization and improves blood flow in sickle cell disease
Melanie Demers,Sarah Sturtevant,Kevin R. Guertin,Dipti Gupta,Kunal Desai,Benjamin F. Vieira,Wenjing Li,Alexandra Hicks,Ayman Ismail,Bronner P. Gonçalves,Giuseppe Di Caprio,Ethan Schonbrun,Scott Hansen,Faik N. Musayev,Martin K. Safo,David K. Wood,John M. Higgins,David R. Light
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Supplemental data
Characterization and statistical modeling of glycosylation changes in sickle cell disease
Heather E. Ashwood,Christopher Ashwood,Anna P. Schmidt,Rebekah L. Gundry,Karin M. Hoffmeister,Waseem Q. Anani
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Supplemental data

THROMBOSIS AND HEMOSTASIS

Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with preexisting anti-FIX immunity
Jocelyn A. Schroeder,Juan Chen,Yingyu Chen,Yuanhua Cai,Hongyin Yu,Jeremy G. Mattson,Paul E. Monahan,Qizhen Shi
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Supplemental data
Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants
Benjamin J. Samelson-Jones,Jonathan D. Finn,Leslie J. Raffini,Elizabeth P. Merricks,Rodney M. Camire,Timothy C. Nichols,Valder R. Arruda
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Supplemental data
Cost-effectiveness of magnetic resonance imaging for diagnosing recurrent ipsilateral deep vein thrombosis
Clinical Trials & Observations
Lisette F. van Dam,Wilbert B. van den Hout,Gargi Gautam,Charlotte E. A. Dronkers,Waleed Ghanima,Jostein Gleditsch,Anders von Heijne,Herman M. A. Hofstee,Marcel M. C. Hovens,Menno V. Huisman,Stan Kolman,Albert T. A. Mairuhu,Mathilde Nijkeuter,Marcel A. van de Ree,Cornelis J. van Rooden,Robin E. Westerbeek,Jan Westerink,Eli Westerlund,Lucia J. M. Kroft,Frederikus A. Klok
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Supplemental data
Outcomes of a clinician-directed protocol for discontinuation of complement inhibition therapy in atypical hemolytic uremic syndrome
Clinical Trials & Observations
Shruti Chaturvedi,Noor Dhaliwal,Sarah Hussain,Kathryn Dane,Harshvardhan Upreti,Evan M. Braunstein,Xuan Yuan,C. John Sperati,Alison R. Moliterno,Robert A. Brodsky
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Population pharmacokinetics of the von Willebrand factor–factor VIII interaction in patients with von Willebrand disease
Laura H. Bukkems,Jessica M. Heijdra,Nico C. B. de Jager,Hendrika C. A. M. Hazendonk,Karin Fijnvandraat,Karina Meijer,Jeroen C. J. Eikenboom,Britta A. P. Laros-van Gorkom,Frank W. G. Leebeek,Marjon H. Cnossen,Ron A. A. Mathôt,for the OPTI-CLOT Study Group
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Supplemental data

TRANSPLANTATION

Haploidentical age-adapted myeloablative transplant and regulatory and effector T cells for acute myeloid leukemia
Clinical Trials & Observations
Antonio Pierini,Loredana Ruggeri,Alessandra Carotti,Franca Falzetti,Simonetta Saldi,Adelmo Terenzi,Claudio Zucchetti,Gianluca Ingrosso,Tiziana Zei,Roberta Iacucci Ostini,Sara Piccinelli,Samanta Bonato,Sara Tricarico,Antonella Mancusi,Sara Ciardelli,Roberto Limongello,Mara Merluzzi,Mauro Di Ianni,Rita Tognellini,Olivia Minelli,Cristina Mecucci,Maria Paola Martelli,Brunangelo Falini,Massimo Fabrizio Martelli,Cynthia Aristei,Andrea Velardi
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Supplemental data
Steroid-dependent acute GVHD after allogeneic hematopoietic cell transplantation: risk factors and clinical outcomes
Clinical Trials & Observations
Najla El Jurdi,Ahmad Rayes,Margaret L. MacMillan,Shernan G. Holtan,Todd E. DeFor,Judith Witte,Mukta Arora,Jo-Anne Young,Daniel J. Weisdorf
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Supplemental data
Relationship of donor age and relationship to outcomes of haploidentical transplantation with posttransplant cyclophosphamide
Clinical Trials & Observations
Amy E. DeZern,Clio Franklin,Hua-Ling Tsai,Phil Hollingsworth Imus,Kenneth R. Cooke,Ravi Varadhan,Richard J. Jones
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Supplemental data
First-in-human phase 1 trial of induced regulatory T cells for graft-versus-host disease prophylaxis in HLA-matched siblings
Clinical Trials & Observations
Margaret L. MacMillan,Keli L. Hippen,David H. McKenna,Diane Kadidlo,Darin Sumstad,Todd E. DeFor,Claudio G. Brunstein,Shernan G. Holtan,Jeffrey S. Miller,Erica D. Warlick,Daniel J. Weisdorf,John E. Wagner,Bruce R. Blazar
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Supplemental data

COMMENTARY

Early detection of treatment failure and early rescue intervention in multiple myeloma: time for new approaches
Clinical Trials & Observations
Juan José Lahuerta,Bruno Paiva,Ana Jiménez de Ubieto,José Sánchez-Pina,María-Victoria Mateos,Joan Bladé,Jesús F. San-Miguel
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  • Cover Image

    Cover Image

    issue cover

    COVER FIGURE
    Ribbon rendering of partial structure of wild-type factor IXa (Protein Data Bank code 1RFN) showing the protease domain in the standard orientation. R338 is highlighted in blue. The amino acids of the catalytic triad (H221, D269, and S365) that form the enzymatic active site are shown in green. See the article by Samelson-Jones et al.

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Wnt inhibitors reduce the unfolded protein response and enhance bortezomib-induced cell death in multiple myeloma
FBXW7β isoform drives transcriptional activation of the proinflammatory TNF cluster in human pro-B cells
Coping in caregivers of patients with hematologic malignancies undergoing hematopoietic stem cell transplantation
Nelarabine combination therapy for relapsed or refractory T-cell acute lymphoblastic lymphoma/leukemia
Posttransplantation cyclophosphamide expands functional myeloid-derived suppressor cells and indirectly influences Tregs

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