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Volume 3, Issue 24

December 23 2019

In this Issue

DRUG ADVANCES
STIMULUS REPORT
CLINICAL TRIALS AND OBSERVATIONS
GENE THERAPY
HEMATOPOIESIS AND STEM CELLS
LYMPHOID NEOPLASIA
MYELOID NEOPLASIA
PLATELETS AND THROMBOPOIESIS
THROMBOSIS AND HEMOSTASIS
TRANSPLANTATION

DRUG ADVANCES

Venetoclax for AML: changing the treatment paradigm
Clinical Trials & Observations

Daniel A. Pollyea,Maria Amaya,Paolo Strati,Marina Y. Konopleva

View Articletitled, Venetoclax for AML: changing the treatment paradigm

STIMULUS REPORT

Primary central nervous system lymphoma: initial features, outcome, and late effects in 75 children and adolescents
Clinical Trials & Observations

Andishe Attarbaschi,Oussama Abla,Leila Ronceray,Shweta Bansil,Simon Bomken,Birgit Burkhardt,Francesco Ceppi,Alan K. S. Chiang,Hema Dave,Alina Fedorova,Michael Henry,Janez Jazbec,Edita Kabickova,Rishi S. Kotecha,Jelena Lazic,Jan Loeffen,Natalia Miakova,Tomoo Osumi,Marta Pillon,Apostolos Pourtsidis,Charlotte Rigaud,Gevorg Tamamyan,Sneha Tandon,Anne Uyttebroeck,Jamie Verdu-Amoros,Wilhelm Woessmann,Grazyna Wrobel,Lisa Giulino-Roth,on behalf of the European Intergroup for Childhood Non-Hodgkin’s Lymphoma, the International Berlin-Frankfurt-Münster Study Group, and selected North American Centers of Pediatric Hematology and Oncology

View Articletitled, Primary central nervous system lymphoma: initial features, outcome, and late effects in 75 children and adolescents

Supplemental data

CLINICAL TRIALS AND OBSERVATIONS

How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients?
Clinical Trials & Observations

Francine Garnache-Ottou,Chrystelle Vidal,Sabeha Biichlé,Florian Renosi,Eve Poret,Maïder Pagadoy,Maxime Desmarets,Anne Roggy,Estelle Seilles,Lou Soret,Françoise Schillinger,Sandrine Puyraimond,Tony Petrella,Claude Preudhomme,Christophe Roumier,Elisabeth A. MacIntyre,Véronique Harrivel,Yohan Desbrosses,Bérengère Gruson,Franck Geneviève,Sylvain Thepot,Yuriy Drebit,Thibaut Leguay,François-Xavier Gros,Nicolas Lechevalier,Pascale Saussoy,Véronique Salaun,Edouard Cornet,Zehaira Benseddik,Richard Veyrat-Masson,Orianne Wagner-Ballon,Célia Salanoubat,Marc Maynadié,Julien Guy,Denis Caillot,Marie-Christine Jacob,Jean-Yves Cahn,Rémy Gressin,Johann Rose,Bruno Quesnel,Estelle Guerin,Franck Trimoreau,Jean Feuillard,Marie-Pierre Gourin,Adriana Plesa,Lucile Baseggio,Isabelle Arnoux,Norbert Vey,Didier Blaise,Romaric Lacroix,Christine Arnoulet,Blandine Benet,Véronique Dorvaux,Caroline Bret,Bernard Drenou,Agathe Debliquis,Véronique Latger-Cannard,Caroline Bonmati,Marie-Christine Bene,Pierre Peterlin,Michel Ticchioni,Pierre-Simon Rohrlich,Anne Arnaud,Stefan Wickenhauser,Valérie Bardet,Sabine Brechignac,Benjamin Papoular,Victoria Raggueneau,Jacques Vargaftig,Rémi Letestu,Daniel Lusina,Thorsten Braun,Vincent Foissaud,Jérôme Tamburini,Hind Bennani,Nicolas Freynet,Catherine Cordonnier,Magali Le Garff-Tavernier,Nathalie Jacques,Karim Maloum,Damien Roos-Weil,Didier Bouscary,Vahid Asnafi,Ludovic Lhermitte,Felipe Suarez,Etienne Lengline,Frédéric Féger,Giorgia Battipaglia,Mohamad Mohty,Sabrina Bouyer,Ouda Ghoual,Elodie Dindinaud,Caroline Basle,Mathieu Puyade,Carinne Lafon,Thierry Fest,Mikael Roussel,Xavier Cahu,Elsa Bera,Sylvie Daliphard,Fabrice Jardin,Lydia Campos,Françoise Solly,Denis Guyotat,Anne-Cécile Galoisy,Alice Eischen,Caroline Mayeur-Rousse,Blandine Guffroy,Christian Recher,Marie Loosveld,Alice Garnier,Vincent Barlogis,Maria Alessandra Rosenthal,Sophie Brun,Nathalie Contentin,Sébastien Maury,Mary Callanan,Christine Lefebvre,Natacha Maillard,Patricia Okamba,Christophe Ferrand,Olivier Adotevi,Philippe Saas,Fanny Angelot-Delettre,Delphine Binda,Eric Deconinck

View Articletitled, How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients?

Supplemental data

GENE THERAPY

Bioengineering hemophilia A–specific microvascular grafts for delivery of full-length factor VIII into the bloodstream

Joseph Neumeyer,Ruei-Zeng Lin,Kai Wang,Xuechong Hong,Tien Hua,Stacy E. Croteau,Ellis J. Neufeld,Juan M. Melero-Martin

View Articletitled, Bioengineering hemophilia A–specific microvascular grafts for delivery of full-length factor VIII into the bloodstream

Supplemental data

HEMATOPOIESIS AND STEM CELLS

Longitudinal assessment of clonal mosaicism in human hematopoiesis via mitochondrial mutation tracking

Caleb A. Lareau,Leif S. Ludwig,Vijay G. Sankaran

View Articletitled, Longitudinal assessment of clonal mosaicism in human hematopoiesis via mitochondrial mutation tracking

Supplemental data

Donor and host coexpressing KIR ligands promote NK education after allogeneic hematopoietic stem cell transplantation

Xiang-Yu Zhao,Xing-Xing Yu,Zheng-Li Xu,Xun-Hong Cao,Ming-Rui Huo,Xiao-Su Zhao,Ying-Jun Chang,Yu Wang,Xiao-Hui Zhang,Lan-Ping Xu,Kai-Yan Liu,Xiao-Jun Huang

View Articletitled, Donor and host coexpressing KIR ligands promote NK education after allogeneic hematopoietic stem cell transplantation

Supplemental data

LYMPHOID NEOPLASIA

Multiple myeloma with 1q21 amplification is highly sensitive to MCL-1 targeting

Anne Slomp,Laura M. Moesbergen,Jia-nan Gong,Marta Cuenca,Peter A. von dem Borne,Pieter Sonneveld,David C. S. Huang,Monique C. Minnema,Victor Peperzak

View Articletitled, Multiple myeloma with 1q21 amplification is highly sensitive to MCL-1 targeting

Supplemental data

Therapy-related acute lymphoblastic leukemia is a distinct entity with adverse genetic features and clinical outcomes
Clinical Trials & Observations

Caner Saygin,Ashwin Kishtagari,Ryan D. Cassaday,Natalie Reizine,Ilana Yurkiewicz,Michaela Liedtke,Wendy Stock,Richard A. Larson,Ross L. Levine,Martin S. Tallman,Jae H. Park,Cassandra Kerr,Bartlomiej Przychodzen,Mikkael A. Sekeres,Matt E. Kalaycio,Hetty E. Carraway,Betty K. Hamilton,Ronald Sobecks,Aaron Gerds,Sudipto Mukherjee,Aziz Nazha,Jaroslaw P. Maciejewski,Anjali S. Advani

View Articletitled, Therapy-related acute lymphoblastic leukemia is a distinct entity with adverse genetic features and clinical outcomes

Supplemental data

MYELOID NEOPLASIA

Retinoic acid synergizes with the unfolded protein response and oxidative stress to induce cell death in FLT3-ITD⁺ AML

Silvia Masciarelli,Ernestina Capuano,Tiziana Ottone,Mariadomenica Divona,Serena Lavorgna,Francesca Liccardo,Martyna Śniegocka,Serena Travaglini,Nelida I. Noguera,Alessandra Picardi,Vincenzo Petrozza,Alessandro Fatica,Luca Tamagnone,Maria Teresa Voso,Francesco Lo Coco,Francesco Fazi

View Articletitled, Retinoic acid synergizes with the unfolded protein response and oxidative stress to induce cell death in FLT3-ITD+ AML

Supplemental data

Atovaquone is active against AML by upregulating the integrated stress pathway and suppressing oxidative phosphorylation

Alexandra M. Stevens,Michael Xiang,Lisa N. Heppler,Isidora Tošić,Kevin Jiang,Jaime O. Munoz,Amos S. Gaikwad,Terzah M. Horton,Xin Long,Padmini Narayanan,Elizabeth L. Seashore,Maci C. Terrell,Raushan Rashid,Michael J. Krueger,Alicia E. Mangubat-Medina,Zachary T. Ball,Pavel Sumazin,Sarah R. Walker,Yoshimasa Hamada,Seiichi Oyadomari,Michele S. Redell,David A. Frank

View Articletitled, Atovaquone is active against AML by upregulating the integrated stress pathway and suppressing oxidative phosphorylation

Supplemental data

The stem cell–specific long noncoding RNA HOXA10-AS in the pathogenesis of KMT2A-rearranged leukemia

Sina Al-Kershi,Raj Bhayadia,Michelle Ng,Lonneke Verboon,Stephan Emmrich,Lucie Gack,Adrian Schwarzer,Till Strowig,Dirk Heckl,Jan-Henning Klusmann

View Articletitled, The stem cell–specific long noncoding RNA HOXA10-AS in the pathogenesis of KMT2A-rearranged leukemia

Supplemental data

Bone marrow oxidative stress and specific antioxidant signatures in myelodysplastic syndromes

Frederic Picou,Christine Vignon,Christelle Debeissat,Sébastien Lachot,Olivier Kosmider,Nathalie Gallay,Amelie Foucault,Marie-Hélène Estienne,Noémie Ravalet,Marie C. Bene,Jorge Domenech,Emmanuel Gyan,Michaela Fontenay,Olivier Herault

View Articletitled, Bone marrow oxidative stress and specific antioxidant signatures in myelodysplastic syndromes

Supplemental data

Managing chronic myeloid leukemia for treatment-free remission: a proposal from the GIMEMA CML WP
Clinical Trials & Observations

Michele Baccarani,Elisabetta Abruzzese,Vincenzo Accurso,Francesco Albano,Mario Annunziata,Sara Barulli,Germana Beltrami,Micaela Bergamaschi,Gianni Binotto,Monica Bocchia,Giovanni Caocci,Isabella Capodanno,Francesco Cavazzini,Michele Cedrone,Marco Cerrano,Monica Crugnola,Mariella D’Adda,Chiara Elena,Carmen Fava,Paola Fazi,Claudio Fozza,Sara Galimberti,Valentina Giai,Antonella Gozzini,Gabriele Gugliotta,Alessandra Iurlo,Gaetano La Barba,Luciano Levato,Alessandro Lucchesi,Luigia Luciano,Francesca Lunghi,Monia Lunghi,Michele Malagola,Roberto Marasca,Bruno Martino,Angela Melpignano,Maria Cristina Miggiano,Enrico Montefusco,Caterina Musolino,Fausto Palmieri,Patrizia Pregno,Davide Rapezzi,Giovanna Rege-Cambrin,Serena Rupoli,Marzia Salvucci,Rosaria Sancetta,Simona Sica,Raffaele Spadano,Fabio Stagno,Mario Tiribelli,Simona Tomassetti,Elena Trabacchi,Massimiliano Bonifacio,Massimo Breccia,Fausto Castagnetti,Fabrizio Pane,Domenico Russo,Giuseppe Saglio,Simona Soverini,Paolo Vigneri,Gianantonio Rosti

View Articletitled, Managing chronic myeloid leukemia for treatment-free remission: a proposal from the GIMEMA CML WP

PLATELETS AND THROMBOPOIESIS

Longitudinal assessments of plasma ADAMTS13 biomarkers predict recurrence of immune thrombotic thrombocytopenic purpura
Clinical Trials & Observations

Jingrui Sui,Wenjing Cao,Konstantine Halkidis,Mohammad S. Abdelgawwad,Nicole K. Kocher,Bryan Guillory,Lance A. Williams,Radhika Gangaraju,Marisa B. Marques,X. Long Zheng

View Articletitled, Longitudinal assessments of plasma ADAMTS13 biomarkers predict recurrence of immune thrombotic thrombocytopenic purpura

Supplemental data

THROMBOSIS AND HEMOSTASIS

von Willebrand factor and factor VIII levels after desmopressin are associated with bleeding phenotype in type 1 VWD
Clinical Trials & Observations

Ferdows Atiq,Lisette M. Schütte,Agnes E. M. Looijen,Johan Boender,Marjon H. Cnossen,Jeroen Eikenboom,Moniek P. M. de Maat,Marieke J. H. A. Kruip,Frank W. G. Leebeek

View Articletitled, von Willebrand factor and factor VIII levels after desmopressin are associated with bleeding phenotype in type 1 VWD

Ibrutinib, but not zanubrutinib, induces platelet receptor shedding of GPIb-IX-V complex and integrin α_IIbβ₃ in mice and humans

Gasim Dobie,Fahd A. Kuriri,Musab M. A. Omar,Fehaid Alanazi,Ali M. Gazwani,Chloe P. S. Tang,Daniel Man-yuen Sze,Sasanka M. Handunnetti,Constantine Tam,Denise E. Jackson

View Articletitled, Ibrutinib, but not zanubrutinib, induces platelet receptor shedding of GPIb-IX-V complex and integrin αIIbβ3 in mice and humans

Supplemental data

TRANSPLANTATION

Vitamin C stabilizes CD8⁺ iTregs and enhances their therapeutic potential in controlling murine GVHD and leukemia relapse

Supinya Iamsawat,Linlu Tian,Anusara Daenthanasanmak,Yongxia Wu,Hung D. Nguyen,David Bastian,Xue-Zhong Yu

View Articletitled, Vitamin C stabilizes CD8+ iTregs and enhances their therapeutic potential in controlling murine GVHD and leukemia relapse

Supplemental data

Allo-HSCT in transplant-naïve patients with Hodgkin lymphoma: a single-arm, multicenter study
Clinical Trials & Observations

Emma Das-Gupta,Kirsty J. Thomson,Adrian J. C. Bloor,Andrew D. Clark,Stephen Mackinnon,Irfan Kayani,Laura Clifton-Hadley,Pip Patrick,Nadjet El-Mehidi,Anthony Lawrie,Amy A. Kirkwood,Nigel H. Russell,David C. Linch,Karl S. Peggs

View Articletitled, Allo-HSCT in transplant-naïve patients with Hodgkin lymphoma: a single-arm, multicenter study

Cover Image

Cover Image

COVER FIGURE
Bioengineered hemophilia A–specific factor VIII (FVIII)–secreting vascular networks in hemophilic mice. Grafts were prepared with human hemophilia A–specific induced pluripotent stem cell–derived endothelial cells that were genetically engineered to overexpress full-length F8. Next, grafts were transplanted subcutaneously into immunodeficient hemophilic mice. After 7 days in vivo, immunofluorescence staining of the explanted grafts revealed the presence of human vessels expressing FVIII (green). Human lumens were stained by h-CD31 (red) and nuclei were stained by DAPI (blue). See the article by Neumeyer et al.
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Table of Contents

DRUG ADVANCES

Venetoclax for AML: changing the treatment paradigm
Clinical Trials & Observations

STIMULUS REPORT

Primary central nervous system lymphoma: initial features, outcome, and late effects in 75 children and adolescents
Clinical Trials & Observations

CLINICAL TRIALS AND OBSERVATIONS

How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients?
Clinical Trials & Observations

GENE THERAPY

Bioengineering hemophilia A–specific microvascular grafts for delivery of full-length factor VIII into the bloodstream

HEMATOPOIESIS AND STEM CELLS

Longitudinal assessment of clonal mosaicism in human hematopoiesis via mitochondrial mutation tracking

Donor and host coexpressing KIR ligands promote NK education after allogeneic hematopoietic stem cell transplantation

LYMPHOID NEOPLASIA

Multiple myeloma with 1q21 amplification is highly sensitive to MCL-1 targeting

Therapy-related acute lymphoblastic leukemia is a distinct entity with adverse genetic features and clinical outcomes
Clinical Trials & Observations

MYELOID NEOPLASIA

Retinoic acid synergizes with the unfolded protein response and oxidative stress to induce cell death in FLT3-ITD⁺ AML

Atovaquone is active against AML by upregulating the integrated stress pathway and suppressing oxidative phosphorylation

The stem cell–specific long noncoding RNA HOXA10-AS in the pathogenesis of KMT2A-rearranged leukemia

Bone marrow oxidative stress and specific antioxidant signatures in myelodysplastic syndromes

Managing chronic myeloid leukemia for treatment-free remission: a proposal from the GIMEMA CML WP
Clinical Trials & Observations

PLATELETS AND THROMBOPOIESIS

Longitudinal assessments of plasma ADAMTS13 biomarkers predict recurrence of immune thrombotic thrombocytopenic purpura
Clinical Trials & Observations

THROMBOSIS AND HEMOSTASIS

von Willebrand factor and factor VIII levels after desmopressin are associated with bleeding phenotype in type 1 VWD
Clinical Trials & Observations

Ibrutinib, but not zanubrutinib, induces platelet receptor shedding of GPIb-IX-V complex and integrin α_IIbβ₃ in mice and humans

TRANSPLANTATION

Vitamin C stabilizes CD8⁺ iTregs and enhances their therapeutic potential in controlling murine GVHD and leukemia relapse

Allo-HSCT in transplant-naïve patients with Hodgkin lymphoma: a single-arm, multicenter study
Clinical Trials & Observations

Email alerts

ASH Publications

American Society of Hematology

Issue Archive

Table of Contents

DRUG ADVANCES

Venetoclax for AML: changing the treatment paradigm Clinical Trials & Observations

STIMULUS REPORT

Primary central nervous system lymphoma: initial features, outcome, and late effects in 75 children and adolescents Clinical Trials & Observations

CLINICAL TRIALS AND OBSERVATIONS

How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients? Clinical Trials & Observations

GENE THERAPY

HEMATOPOIESIS AND STEM CELLS

LYMPHOID NEOPLASIA

Therapy-related acute lymphoblastic leukemia is a distinct entity with adverse genetic features and clinical outcomes Clinical Trials & Observations

MYELOID NEOPLASIA

Managing chronic myeloid leukemia for treatment-free remission: a proposal from the GIMEMA CML WP Clinical Trials & Observations

PLATELETS AND THROMBOPOIESIS

Longitudinal assessments of plasma ADAMTS13 biomarkers predict recurrence of immune thrombotic thrombocytopenic purpura Clinical Trials & Observations

THROMBOSIS AND HEMOSTASIS

von Willebrand factor and factor VIII levels after desmopressin are associated with bleeding phenotype in type 1 VWD Clinical Trials & Observations

TRANSPLANTATION

Allo-HSCT in transplant-naïve patients with Hodgkin lymphoma: a single-arm, multicenter study Clinical Trials & Observations

Cover Image

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only

Venetoclax for AML: changing the treatment paradigm
Clinical Trials & Observations

Primary central nervous system lymphoma: initial features, outcome, and late effects in 75 children and adolescents
Clinical Trials & Observations

How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients?
Clinical Trials & Observations

Therapy-related acute lymphoblastic leukemia is a distinct entity with adverse genetic features and clinical outcomes
Clinical Trials & Observations

Managing chronic myeloid leukemia for treatment-free remission: a proposal from the GIMEMA CML WP
Clinical Trials & Observations

Longitudinal assessments of plasma ADAMTS13 biomarkers predict recurrence of immune thrombotic thrombocytopenic purpura
Clinical Trials & Observations

von Willebrand factor and factor VIII levels after desmopressin are associated with bleeding phenotype in type 1 VWD
Clinical Trials & Observations

Allo-HSCT in transplant-naïve patients with Hodgkin lymphoma: a single-arm, multicenter study
Clinical Trials & Observations