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Issue Archive

Volume 8, Issue 7
April 9 2024
Issue Cover

Table of Contents

INSIDE BLOOD ADVANCES

Long live the red blood cell: biotin tagging in SCD
Clinical Trials & Observations
Sarah McCuskee,Susanna Curtis
View Articletitled, Long live the red blood cell: biotin tagging in SCD
Mending RAG2: gene editing for treatment of RAG2 deficiency
Elizabeth D. Hicks,Michael D. Keller
View Articletitled, Mending RAG2: gene editing for treatment of RAG2 deficiency

DRUG ADVANCES

Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
Clinical Trials & Observations
Xavier M. Anguela,Katherine A. High
View Articletitled, Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec

CLINICAL TRIALS AND OBSERVATIONS

A phase 1-2 trial of DA-EPOCH-R plus ixazomib for MYC-aberrant lymphoid malignancies: the DACIPHOR regimen
Clinical Trials & Observations
Reem Karmali,Carlos Galvez,Mehdi Hamadani,Leo Gordon,Jane Winter,Shuo Ma,Valerie Nelson,Timothy S. Fenske,Nirav N. Shah,Deepa Jagadeesh,Andreas Klein,Irene Helenowski,Ruohui Chen,Xinlei Mi,Adam Petrich,Andrew M. Evens,Barbara Pro
View Articletitled, A phase 1-2 trial of DA-EPOCH-R plus ixazomib for MYC-aberrant lymphoid malignancies: the DACIPHOR regimen
Supplemental data
Biomarker analysis of the ASPEN study comparing zanubrutinib with ibrutinib for patients with Waldenström macroglobulinemia
Clinical Trials & Observations
Constantine S. Tam,Stephen Opat,Shirley D'Sa,Wojciech Jurczak,Hui-Peng Lee,Gavin Cull,Roger G. Owen,Paula Marlton,Björn E. Wahlin,Ramón García-Sanz,Helen McCarthy,Stephen Mulligan,Alessandra Tedeschi,Jorge J. Castillo,Jarosław Czyż,Carlos Fernández De Larrea,David Belada,Edward Libby,Jeffrey Matous,Marina Motta,Tanya Siddiqi,Monica Tani,Marek Trněný,Monique C. Minnema,Christian Buske,Véronique Leblond,Steven P. Treon,Judith Trotman,Binghao Wu,Yiling Yu,Zhirong Shen,Wai Y. Chan,Jingjing Schneider,Heather Allewelt,Aileen Cohen,Meletios A. Dimopoulos
View Articletitled, Biomarker analysis of the ASPEN study comparing zanubrutinib with ibrutinib for patients with Waldenström macroglobulinemia
Supplemental data
Management of acute breakthrough hemolysis with intensive pegcetacoplan dosing in patients with PNH
Clinical Trials & Observations
Morag Griffin,Richard J Kelly,Jens Panse,Carlos de Castro,Jeff Szer,Regina Horneff,Lisa Tan,Michael Yeh,Régis Peffault de Latour
View Articletitled, Management of acute breakthrough hemolysis with intensive pegcetacoplan dosing in patients with PNH
Immunoglobulin replacement vs prophylactic antibiotics for hypogammaglobulinemia secondary to hematological malignancy
Clinical Trials & Observations
Zoe K. McQuilten,Robert Weinkove,Le Thi Phuong Thao,Philip Crispin,Amber Degelia,Claire Dendle,Michael Gilbertson,Anna Johnston,Anastazia Keegan,Dominic Pepperell,Humphrey Pullon,John Reynolds,Tina van Tonder,Judith Trotman,Neil Waters,Cameron Wellard,Helen Weston,C. Orla Morrissey,Erica M. Wood,in collaboration with the Australasian Leukaemia and Lymphoma Group
View Articletitled, Immunoglobulin replacement vs prophylactic antibiotics for hypogammaglobulinemia secondary to hematological malignancy
Supplemental data
In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation
Clinical Trials & Observations
Alexis K. Leonard,Dana Furstenau,Zaina Inam,Christina Luckett,Rebecca Chu,Selami Demirci,Khaled Essawi,Bjorg Gudmundsdottir,Malikiya Hinds,Julia DiNicola,Quan Li,William A. Eaton,Troy Cellmer,Xunde Wang,Swee Lay Thein,Elizabeth R. Macari,Sara VanNest,Matthew M. Hsieh,Melissa Bonner,Francis J. Pierciey,John F. Tisdale
View Articletitled, In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation
Supplemental data

GENE THERAPY

Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
Mara Pavel-Dinu,Cameron L. Gardner,Yusuke Nakauchi,Tomoki Kawai,Ottavia M. Delmonte,Boaz Palterer,Marita Bosticardo,Francesca Pala,Sebastien Viel,Harry L. Malech,Hana Y. Ghanim,Nicole M. Bode,Gavin L. Kurgan,Angela M. Detweiler,Christopher A. Vakulskas,Norma F. Neff,Adam Sheikali,Sherah T. Menezes,Jade Chrobok,Elaine M. Hernández González,Ravindra Majeti,Luigi D. Notarangelo,Matthew H. Porteus
View Articletitled, Genetically corrected <em>RAG2</em>-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
Supplemental data

IMMUNOBIOLOGY AND IMMUNOTHERAPY

Cysteine-binding adjuvant enhances survival and promotes immune function in a murine model of acute myeloid leukemia
Anna J. Slezak,Kevin Chang,Taryn N. Beckman,Kirsten C. Refvik,Aaron T. Alpar,Abigail L. Lauterbach,Ani Solanki,Jung Woo Kwon,Suzana Gomes,Aslan Mansurov,Jeffrey A. Hubbell
View Articletitled, Cysteine-binding adjuvant enhances survival and promotes immune function in a murine model of acute myeloid leukemia
Supplemental data

LYMPHOID NEOPLASIA

Biological signatures of the International Prognostic Index in diffuse large B-cell lymphoma
Yue Wang,Qing Shi,Zi-Yang Shi,Shuang Tian,Mu-Chen Zhang,Rong Shen,Di Fu,Lei Dong,Hong-Mei Yi,Bin-Shen Ouyang,Rong-Ji Mu,Shu Cheng,Li Wang,Peng-Peng Xu,Wei-Li Zhao
View Articletitled, Biological signatures of the International Prognostic Index in diffuse large B-cell lymphoma
Supplemental data
CD8 effector T cells enhance teclistamab response in BCMA-exposed and -naïve multiple myeloma
Ross S. Firestone,Devin McAvoy,Tala Shekarkhand,Edith Serrano,Issam Hamadeh,Alice Wang,Menglei Zhu,Wei Ge Qin,Dhwani Patel,Carlyn R. Tan,Malin Hultcrantz,Sham Mailankody,Hani Hassoun,Urvi S. Shah,Neha Korde,Kylee H. Maclachlan,Heather J. Landau,Michael Scordo,Gunjan L. Shah,Oscar B. Lahoud,Sergio Giralt,Kazunori Murata,Kinga K. Hosszu,David J. Chung,Alexander M. Lesokhin,Saad Z. Usmani
View Articletitled, CD8 effector T cells enhance teclistamab response in BCMA-exposed and -naïve multiple myeloma
Supplemental data
Mass spectrometry-detected MGUS is associated with obesity and other novel modifiable risk factors in a high-risk population
David J. Lee,Habib El-Khoury,Angela C. Tramontano,Jean-Baptiste Alberge,Jacqueline Perry,Maya I. Davis,Erica Horowitz,Robert Redd,Dhananjay Sakrikar,David Barnidge,Mark C. Perkins,Stephen Harding,Lorelei Mucci,Timothy R. Rebbeck,Irene M. Ghobrial,Catherine R. Marinac
View Articletitled, Mass spectrometry-detected MGUS is associated with obesity and other novel modifiable risk factors in a high-risk population
Supplemental data

MYELOID NEOPLASIA

Monosomy 7/del(7q) cause sensitivity to inhibitors of nicotinamide phosphoribosyltransferase in acute myeloid leukemia
Samuli Eldfors,Joseph Saad,Nemo Ikonen,Disha Malani,Markus Vähä-Koskela,Bjørn T. Gjertsen,Mika Kontro,Kimmo Porkka,Caroline A. Heckman
View Articletitled, Monosomy 7/del(7q) cause sensitivity to inhibitors of nicotinamide phosphoribosyltransferase in acute myeloid leukemia
Supplemental data
A real-world analysis of clinical outcomes in AML with myelodysplasia-related changes: a comparison of ICC and WHO-HAEM5 criteria
Qianghua Zhou,Davidson Zhao,Mojgan Zarif,Marta B. Davidson,Mark D. Minden,Anne Tierens,Yu Wing Tony Yeung,Cuihong Wei,Hong Chang
View Articletitled, A real-world analysis of clinical outcomes in AML with myelodysplasia-related changes: a comparison of ICC and WHO-HAEM5 criteria
Supplemental data

PHAGOCYTES, GRANULOCYTES, AND MYELOPOIESIS

Impact of different genetic mutations on granulocyte development and G-CSF responsiveness in congenital neutropenia
Xin Meng,Hai Zhang,Lulu Dong,Qing Min,Meiping Yu,Yaxuan Li,Lipin Liu,Wenjie Wang,Wenjing Ying,Jinqiao Sun,Ji-Yang Wang,Jia Hou,Xiaochuan Wang
View Articletitled, Impact of different genetic mutations on granulocyte development and G-CSF responsiveness in congenital neutropenia
Supplemental data

PLATELETS AND THROMBOPOIESIS

Altered platelet-megakaryocyte endocytosis and trafficking of albumin and fibrinogen in RUNX1 haplodeficiency
Fabiola Del Carpio-Cano,Guangfen Mao,Lawrence E. Goldfinger,Jeremy Wurtzel,Liying Guan,Mohammad Afaque Alam,Kiwon Lee,Mortimer Poncz,A. Koneti Rao
View Articletitled, Altered platelet-megakaryocyte endocytosis and trafficking of albumin and fibrinogen in <em>RUNX1</em> haplodeficiency
Supplemental data
Long-term treatment with rilzabrutinib in patients with immune thrombocytopenia
Clinical Trials & Observations
David J. Kuter,Jiri Mayer,Merlin Efraim,Lachezar H. Bogdanov,Ross Baker,Zane Kaplan,Mamta Garg,Marek Trněný,Philip Y. Choi,A. J. Gerard Jansen,Vickie McDonald,Robert Bird,Jaromir Gumulec,Milan Kostal,Terry Gernsheimer,Waleed Ghanima,Ahmed Daak,Nichola Cooper
View Articletitled, Long-term treatment with rilzabrutinib in patients with immune thrombocytopenia
Supplemental data

RED CELLS, IRON, AND ERYTHROPOIESIS

Deciphering the regulatory landscape of murine splenic response to anemic stress at single-cell resolution
Chong Yang,Rui Yokomori,Lee Hui Chua,Shi Hao Tan,Mun Yee Koh,Haruhito Totani,Takaomi Sanda,Toshio Suda
View Articletitled, Deciphering the regulatory landscape of murine splenic response to anemic stress at single-cell resolution
Supplemental data

THROMBOSIS AND HEMOSTASIS

Type 2M/2A von Willebrand disease: a shared phenotype between type 2M and 2A
Omid Seidizadeh,Luca Mollica,Serena Zambarbieri,Luciano Baronciani,Andrea Cairo,Paola Colpani,Giovanna Cozzi,Maria Teresa Pagliari,Alessandro Ciavarella,Simona M. Siboni,Flora Peyvandi
View Articletitled, Type 2M/2A von Willebrand disease: a shared phenotype between type 2M and 2A
Supplemental data

TRANSFUSION MEDICINE

Aberrant GPA expression and regulatory function of red blood cells in sickle cell disease
Juliana N. Marshall,Matthew N. Klein,Pratap Karki,Kamoltip Promnares,Saini Setua,Xiaoxuan Fan,Paul W. Buehler,Konstantin G. Birukov,Gerardo R. Vasta,Magali J. Fontaine
View Articletitled, Aberrant GPA expression and regulatory function of red blood cells in sickle cell disease
Supplemental data

RESEARCH LETTERS

Regression of smoldering myeloma with treatment of Gaucher disease
Kevin Barley,Anshuman Parekh,Syed Salam,Damodara Rao Mendu,Ravi Prakash Shukla,Deepa Vatti,Daniel Verina,Chanan Stauffer,Christian Salib,Siraj El Jamal,Julie Teruya-Feldstein,Amy S. Duffield,Violetta V. Leshchenko,Sundar Jagannath,Manisha Balwani,Samir Parekh
View Articletitled, Regression of smoldering myeloma with treatment of Gaucher disease
Supplemental data
Efficacy and safety of daratumumab in pure red cell aplasia after allogeneic transplantation: Dutch real-world data
Clinical Trials & Observations
Flores Weverling,Mieke Roeven,Clara Nijssen,Annoek E. C. Broers,Elisabeth Dovern,Anna van Rhenen,Geerte van Sluis,Carin L. E. Hazenberg,Peter van Balen,Maria T. Kuipers,Karen M. K. de Vooght,Linde Morsink,Jürgen Kuball,Erfan Nur,Moniek A. de Witte
View Articletitled, Efficacy and safety of daratumumab in pure red cell aplasia after allogeneic transplantation: Dutch real-world data
Supplemental data
Impact of MYC and BCL2 double expression on outcomes in primary CNS lymphoma: a UK multicenter analysis
Edward Poynton,Emily Chernucha,James Day,Catherine Prodger,David Hopkins,Pallav Rakesh,Tess O’Neill,Nisha Thakrar,Ayse Akarca,Esraa Jamal,Ayesha Ali,Amy A. Kirkwood,Sabine Pomplun,Teresa Marafioti,Maria Calaminici,Paul Greaves,Sridhar Chaganti,Pam McKay,Jeffery Smith,Toby A. Eyre,Nicolas Martinez-Calle,Kate Cwynarski,Christopher P. Fox,Jessica Okosun
View Articletitled, Impact of MYC and BCL2 double expression on outcomes in primary CNS lymphoma: a UK multicenter analysis
Supplemental data

ERRATUM

Matsui H, Arai Y, Imoto H, et al. Risk factors and appropriate therapeutic strategies for thrombotic microangiopathy after allogeneic HSCT. Blood Adv. 2020;4(13):3169-3179.
View Articletitled, Matsui H, Arai Y, Imoto H, et al. Risk factors and appropriate therapeutic strategies for thrombotic microangiopathy after allogeneic HSCT. <em>Blood Adv.</em> 2020;4(13):3169-3179.
  • Cover Image

    Cover Image

    issue cover

    A representative image of the effect of knockdown of RUNX1 and CAV1 on fibrinogen–Alexa 647 (red) uptake and colocalization with RAB11 (green), a marker for recycling endosomes, in phorbol 12-myristate 13-acetate (PMA)–treated human erythroleukemia cells. Cell suspensions (control cells and those with small interfering RNA knockdown of RUNX1 and CAV1) were incubated with fibrinogen–Alexa 647 for 30 minutes, fixed, and immobilized on poly-l-lysine-coated coverslips. See the article by Del Carpio-Cano et al.

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