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Volume 8, Issue 6

March 26 2024

In this Issue

INSIDE BLOOD ADVANCES
CLINICAL TRIALS AND OBSERVATIONS
GENE THERAPY
HEALTH SERVICES AND OUTCOMES
IMMUNOBIOLOGY AND IMMUNOTHERAPY
LYMPHOID NEOPLASIA
MYELOID NEOPLASIA
PLATELETS AND THROMBOPOIESIS
RED CELLS, IRON, AND ERYTHROPOIESIS
THROMBOSIS AND HEMOSTASIS
TRANSFUSION MEDICINE
TRANSPLANTATION
RESEARCH LETTERS
COMMENTARY
ERRATUM

INSIDE BLOOD ADVANCES

Platelets, plasma, and proteostasis: a translation tightrope

Irina Portier,Matthew T. Rondina

View Articletitled, Platelets, plasma, and proteostasis: a translation tightrope

Navigating the prognostic role of transfusions after CAR-T

Mohammad Alhomoud,Kai Rejeski

View Articletitled, Navigating the prognostic role of transfusions after CAR-T

CLINICAL TRIALS AND OBSERVATIONS

Development of an umbilical cord blood transplantation–specific nonrelapse mortality risk assessment score
Clinical Trials & Observations

Yosuke Okada,Yoshiaki Usui,Hiromi Hayashi,Masashi Nishikubo,Tomomi Toubai,Naoyuki Uchida,Masatsugu Tanaka,Makoto Onizuka,Satoshi Takahashi,Noriko Doki,Yasufumi Uehara,Yumiko Maruyama,Kazuya Ishiwata,Toshiro Kawakita,Masashi Sawa,Tetsuya Eto,Fumihiko Ishimaru,Koji Kato,Takahiro Fukuda,Yoshiko Atsuta,Junya Kanda,Kimikazu Yakushijin,Hideki Nakasone

View Articletitled, Development of an umbilical cord blood transplantation–specific nonrelapse mortality risk assessment score

Supplemental data

von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease
Clinical Trials & Observations

Robert F. Sidonio, Jr.,Ana Boban,Leonid Dubey,Adlette Inati,Csongor Kiss,Zoltan Boda,Toshko Lissitchkov,Laszlo Nemes,Dzmitry Novik,Elina Peteva,Ali T. Taher,Margarita Arkadevna Timofeeva,Kateryna V. Vilchevska,Vladimir Vdovin,Sylvia Werner,Sigurd Knaub,Claudia Djambas Khayat

View Articletitled, von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease

Supplemental data

A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial
Clinical Trials & Observations

Manuel Carcao,Michele Schiavulli,Roshni Kulkarni,Pablo Rendo,Meredith Foster,Elena Santagostino,Sandra Casiano,Christoph Königs

View Articletitled, A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial

Supplemental data

Allogeneic hematopoietic cell transplantation for adult metachromatic leukodystrophy: a case series
Clinical Trials & Observations

Andreas Riedel,Christoph Faul,Kristina Reuss,Jan C. Schröder,Peter J. Lang,Claudia Lengerke,Nadine Weissert,Holger Hengel,Samuel Gröschel,Ludger Schoels,Wolfgang A. Bethge

View Articletitled, Allogeneic hematopoietic cell transplantation for adult metachromatic leukodystrophy: a case series

GENE THERAPY

Clinical features of neurotoxicity after CD19 CAR T-cell therapy in mantle cell lymphoma

Esther H. Nie,Yi-Jiun Su,John H. Baird,Neha Agarwal,Sushma Bharadwaj,Wen-Kai Weng,Melody Smith,Saurabh Dahiya,May H. Han,Jeffrey E. Dunn,Lucas B. Kipp,David B. Miklos,Brian J. Scott,Matthew J. Frank

View Articletitled, Clinical features of neurotoxicity after CD19 CAR T-cell therapy in mantle cell lymphoma

Supplemental data

HEALTH SERVICES AND OUTCOMES

Coping in patients with hematologic malignancies undergoing hematopoietic cell transplantation

Richard Newcomb,Hermioni L. Amonoo,Ashley M. Nelson,Joanna Choe,Katherine Holmbeck,Anisa Nabily,Stephanie J. Lee,Thomas W. LeBlanc,Areej El-Jawahri

View Articletitled, Coping in patients with hematologic malignancies undergoing hematopoietic cell transplantation

Supplemental data

IMMUNOBIOLOGY AND IMMUNOTHERAPY

Transfusion needs after CAR T-cell therapy for large B-cell lymphoma: predictive factors and outcome (a DESCAR-T study)

Samuel Vic,Jean-Baptiste Thibert,Emmanuel Bachy,Guillaume Cartron,Thomas Gastinne,Franck Morschhauser,Fabien Le Bras,Kamal Bouabdallah,Fabien Despas,Jacques-Olivier Bay,Marie-Thérèse Rubio,Mohamad Mohty,Olivier Casasnovas,Sylvain Choquet,Cristina Castilla-Llorente,Stéphanie Guidez,Michaël Loschi,Blandine Guffroy,Sylvain Carras,Laurianne Drieu La Rochelle,Mathilde Guillet,Roch Houot

View Articletitled, Transfusion needs after CAR T-cell therapy for large B-cell lymphoma: predictive factors and outcome (a DESCAR-T study)

Supplemental data

Navigating the prognostic role of transfusions after CAR-T

Mohammad Alhomoud,Kai Rejeski

View Articletitled, Navigating the prognostic role of transfusions after CAR-T

LYMPHOID NEOPLASIA

Reprogramming of pyrimidine nucleotide metabolism supports vigorous cell proliferation of normal and malignant T cells

Tatsuro Watanabe,Yuta Yamamoto,Yuki Kurahashi,Kazunori Kawasoe,Keisuke Kidoguchi,Hiroshi Ureshino,Kazuharu Kamachi,Nao Yoshida-Sakai,Yuki Fukuda-Kurahashi,Hideaki Nakamura,Seiji Okada,Eisaburo Sueoka,Shinya Kimura

View Articletitled, Reprogramming of pyrimidine nucleotide metabolism supports vigorous cell proliferation of normal and malignant T cells

Supplemental data

IELSG30 phase 2 trial: intravenous and intrathecal CNS prophylaxis in primary testicular diffuse large B-cell lymphoma
Clinical Trials & Observations

Annarita Conconi,Annalisa Chiappella,Andrés J. M. Ferreri,Anastasios Stathis,Barbara Botto,Marianna Sassone,Gianluca Gaidano,Monica Balzarotti,Francesco Merli,Alessandra Tucci,Anna Vanazzi,Monica Tani,Riccardo Bruna,Lorella Orsucci,Maria Giuseppina Cabras,Melania Celli,Ombretta Annibali,Anna Marina Liberati,Manuela Zanni,Chiara Ghiggi,Francesco Pisani,Graziella Pinotti,Fausto Dore,Fabiana Esposito,Maria Cristina Pirosa,Marina Cesaretti,Luisella Bonomini,Umberto Vitolo,Emanuele Zucca,on behalf of the International Extranodal Lymphoma Study Group

View Articletitled, IELSG30 phase 2 trial: intravenous and intrathecal CNS prophylaxis in primary testicular diffuse large B-cell lymphoma

MYELOID NEOPLASIA

Phase 2 study of add-on parsaclisib for patients with myelofibrosis and suboptimal response to ruxolitinib: final results
Clinical Trials & Observations

Abdulraheem Yacoub,Uma Borate,Raajit K. Rampal,Haris Ali,Eunice S. Wang,Aaron T. Gerds,Gabriela Hobbs,Marina Kremyanskaya,Elliott Winton,Casey O’Connell,Swati Goel,Stephen T. Oh,Gary Schiller,James McCloskey,Jeanne Palmer,Houston Holmes,Steven Hager,Albert Assad,Susan Erickson-Viitanen,Feng Zhou,Naval Daver

View Articletitled, Phase 2 study of add-on parsaclisib for patients with myelofibrosis and suboptimal response to ruxolitinib: final results

Supplemental data

PLATELETS AND THROMBOPOIESIS

Prednisone vs high-dose dexamethasone in newly diagnosed adult primary immune thrombocytopenia: a randomized trial
Clinical Trials & Observations

Maria Gabriella Mazzucconi,Francesco Rodeghiero,Giuseppe Avvisati,Valerio De Stefano,Luigi Gugliotta,Marco Ruggeri,Nicola Vianelli,Paola Fazi,Francesca Paoloni,Valeria Sargentini,Erminia Baldacci,Antonietta Ferretti,Bruno Martino,Iolanda Donatella Vincelli,Giuseppe Carli,Stefania Fortuna,Mauro Di Ianni,Paola Ranalli,Francesca Palandri,Nicola Polverelli,Elisabetta Lugli,Elena Rivolti,Andrea Patriarca,Angela Rago,Mariella D’Adda,Massimo Gentile,Sergio Siragusa,Silvia Sibilla,Angelo Michele Carella,Elena Rossi,Roberta Battistini,Francesco Zaja,Monica Bocchia,Nicola Di Renzo,Pellegrino Musto,Monica Crugnola,Anna Chiara Giuffrida,Mauro Krampera,Agostino Tafuri,Cristina Santoro

View Articletitled, Prednisone vs high-dose dexamethasone in newly diagnosed adult primary immune thrombocytopenia: a randomized trial

RED CELLS, IRON, AND ERYTHROPOIESIS

Modeling primitive and definitive erythropoiesis with induced pluripotent stem cells

Giulia Pavani,Joshua G. Klein,Catriana C. Nations,Jonathan H. Sussman,Kai Tan,Hyun Hyung An,Osheiza Abdulmalik,Christopher S. Thom,Peter A. Gearhart,Camryn M. Willett,Jean Ann Maguire,Stella T. Chou,Deborah L. French,Paul Gadue

View Articletitled, Modeling primitive and definitive erythropoiesis with induced pluripotent stem cells

Supplemental data

THROMBOSIS AND HEMOSTASIS

Congenital fibrinogen disorders: a retrospective clinical and genetic analysis of the Prospective Rare Bleeding Disorders Database

Samin Mohsenian,Roberta Palla,Marzia Menegatti,Andrea Cairo,Anna Lecchi,Alessandro Casini,Marguerite Neerman-Arbez,Rosanna Asselta,Sara Scardo,Simona Maria Siboni,Jan Blatny,Ondrej Zapletal,Jean-Francois Schved,Muriel Giansily-Blaizot,Susan Halimeh,Mohamad Ayman Daoud,Helen Platokouki,Helen Pergantou,Roger E. G. Schutgens,Monique Van Haaften-Spoor,Paul Brons,Britta Laros-van Gorkom,Elise Van Pinxten,Munira Borhany,Naveena Fatima,Danijela Mikovic,Marko Saracevic,Gül Nihal Özdemir,Yılmaz Ay,Michael Makris,Caryl Lockley,Andrew Mumford,Andrew Harvey,Steve Austin,Amy Shapiro,Adrianna Williamson,Catherine McGuinn,Ilene Goldberg,Philippe De Moerloose,Flora Peyvandi

View Articletitled, Congenital fibrinogen disorders: a retrospective clinical and genetic analysis of the Prospective Rare Bleeding Disorders Database

Plasma growth factors maintain constitutive translation in platelets to regulate reactivity and thrombotic potential

Jeremy G. T. Wurtzel,Sophia Lazar,Shayan Askari,Xuefei Zhao,Jenna Severa,Francis Ayombil,James V. Michael,Rodney M. Camire,Steven E. McKenzie,Timothy J. Stalker,Peisong Ma,Lawrence E. Goldfinger

View Articletitled, Plasma growth factors maintain constitutive translation in platelets to regulate reactivity and thrombotic potential

Supplemental data

Platelets, plasma, and proteostasis: a translation tightrope

Irina Portier,Matthew T. Rondina

View Articletitled, Platelets, plasma, and proteostasis: a translation tightrope

TRANSFUSION MEDICINE

CEBPA double mutations associated with ABO antigen weakness in hematologic diseases

Seung Jun Choi,Hyun Kyung Kim,Eun Jung Suh,Soon Sung Kwon,Saeam Shin,Seung-Tae Lee,Sinyoung Kim

View Articletitled, CEBPA double mutations associated with ABO antigen weakness in hematologic diseases

Supplemental data

TRANSPLANTATION

A multicenter study of posttransplantation low-dose inotuzumab ozogamicin to prevent relapse of acute lymphoblastic leukemia
Clinical Trials & Observations

Leland L. Metheny,Ronald Sobecks,Christina Cho,Pingfu Fu,Seunghee Margevicius,Jiasheng Wang,Lisa Ciarrone,Shelby Kopp,Robin D. Convents,Navneet Majhail,Paolo F. Caimi,Folashade Otegbeye,Brenda W. Cooper,Molly Gallogly,Ehsan Malek,Benjamin Tomlinson,Aaron T. Gerds,Betty Hamilton,Sergio Giralt,Miguel-Angel Perales,Marcos de Lima

View Articletitled, A multicenter study of posttransplantation low-dose inotuzumab ozogamicin to prevent relapse of acute lymphoblastic leukemia

Supplemental data

Liver abnormalities are frequent and persistent in patients with Fanconi anemia
Clinical Trials & Observations

Alana J. Snyder,Kathleen M. Campbell,Adam Lane,Parinda A. Mehta,Kasiani Myers,Stella M. Davies,Jane Koo

View Articletitled, Liver abnormalities are frequent and persistent in patients with Fanconi anemia

RESEARCH LETTERS

Targeting CXCR4, VLA-4, and CXCR2 for hematopoietic stem cell mobilization

Daniel Cancilla,Michael P. Rettig,Darja Karpova,Haresh Thakellapalli,Megh Singh,Marvin J. Meyers,Peter G. Ruminski,Stephanie Christ,Ezhilarasi Chendamarai,Feng Gao,Leah Gehrs,Julie K. Ritchey,Michael Prinsen,John F. DiPersio

View Articletitled, Targeting CXCR4, VLA-4, and CXCR2 for hematopoietic stem cell mobilization

Supplemental data

Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study
Clinical Trials & Observations

Kinuko Mitani,Jong Wook Lee,Jun Ho Jang,Yoshiaki Tomiyama,Koji Miyazaki,Koji Nagafuji,Kensuke Usuki,Nobuhiko Uoshima,Tomoaki Fujisaki,Hiroshi Kosugi,Itaru Matsumura,Ko Sasaki,Masahiro Kizaki,Masashi Sawa,Michihiro Hidaka,Naoki Kobayashi,Satoshi Ichikawa,Yuji Yonemura,Kenta Murotani,Mami Shimizu,Akira Matsuda,Keiya Ozawa,Shinji Nakao

View Articletitled, Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study

Supplemental data

Longitudinal plasma proteomics in CAR T–cell therapy patients implicates neutrophils and NETosis in the genesis of CRS
Clinical Trials & Observations

Christopher Flora,Mary Olesnavich,Yu Zuo,Erin Sandford,Rashmi Madhukar,Michelle Rozwadowski,Kavya Sugur,Andrew Ly,Ata Alpay Canbaz,Audra Shedeck,Gen Li,Marcus J. Geer,Gregory A. Yanik,Monalisa Ghosh,David G. Frame,Challice L. Bonifant,Tania Jain,Jason S. Knight,Sung Won Choi,Muneesh Tewari

View Articletitled, Longitudinal plasma proteomics in CAR T–cell therapy patients implicates neutrophils and NETosis in the genesis of CRS

Supplemental data

Venetoclax-rituximab is active in patients with BTKi-exposed CLL, but durable treatment-free remissions are uncommon

Thomas E. Lew,Rory Bennett,Victor S. Lin,Ashley Whitechurch,Sasanka M. Handunnetti,Paula Marlton,Yandong Shen,Stephen P. Mulligan,Joshua Casan,Piers Blombery,Constantine S. Tam,Andrew W. Roberts,John F. Seymour,Philip A. Thompson,Mary A. Anderson

View Articletitled, Venetoclax-rituximab is active in patients with BTKi-exposed CLL, but durable treatment-free remissions are uncommon

Supplemental data

Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT

Carmelo Gurnari,Linda Koster,Laurien Baaij,Mael Heiblig,Ibrahim Yakoub-Agha,Matthew Collin,Jakob Passweg,Claude E. Bulabois,Anjum Khan,Michael Loschi,Fabrizio Carnevale-Schianca,Elena Crisà,Daniela Caravelli,Jürgen Kuball,Francesco Saraceni,Attilio Olivieri,Alessandro Rambaldi,Austin G. Kulasekararaj,Patrick J. Hayden,Manuela Badoglio,Francesco Onida,Christof Scheid,Franco Franceschini,Arsène Mekinian,Sinisa Savic,Maria Teresa Voso,Joanna Drozd-Sokolowska,John A Snowden,Kavita Raj,Tobias Alexander,Marie Robin,Raffaella Greco,Donal P. McLornan

View Articletitled, Allogeneic hematopoietic cell transplantation for VEXAS syndrome: results of a multicenter study of the EBMT

Supplemental data

Impact of imaging frequency on progression-free survival in Alliance trials enrolling patients with follicular lymphoma
Clinical Trials & Observations

Sarah C. Rutherford,Jun Yin,Levi D. Pederson,Kristie A. Blum,Peter Martin,Sin-Ho Jung,Barbara Grant,Cara Rosenbaum,Bruce D. Cheson,Nancy L. Bartlett,Sumithra J. Mandrekar,John P. Leonard

View Articletitled, Impact of imaging frequency on progression-free survival in Alliance trials enrolling patients with follicular lymphoma

Supplemental data

Self-reported pain levels for emergency department visits associated with sickle cell disease in the United States

Brandon K. Attell,Blake T. McGee,Ann M. DiGirolamo,Lindsey L. Cohen,Angela B. Snyder

View Articletitled, Self-reported pain levels for emergency department visits associated with sickle cell disease in the United States

Large B-cell lymphoma-IRF4+ in children and young people: time to reduce chemotherapy in a rare malignant mature B-cell neoplasm?
Clinical Trials & Observations

Minke Huibers,Oussama Abla,Mara Andrés,Olga Balagué,Auke Beishuizen,Elisa Carraro,Alan Chiang,Monika Csóka,Bianca-Andreea David,Maëlle de Ville de Goyet,Gil Gilad,Daiki Hori,Rishi S. Kotecha,Edita Kabickova,Wolfram Klapper,Natasha Miakova,Veronique Minard-Colin,Atsuko Nakazawa,Marta Pillon,Charlotte Rigaud,Itziar Salaverria,Ida Tölle,Jaime Verdú-Amorós,Hannah von Mersi,Wilhelm Wössmann,Birgit Burkhardt,Andishe Attarbaschi

View Articletitled, Large B-cell lymphoma-IRF4+ in children and young people: time to reduce chemotherapy in a rare malignant mature B-cell neoplasm?

Supplemental data

COMMENTARY

Fine and Gray or Cox model?

Leonardo Javier Arcuri,Fabio Pires de Souza Santos,Guilherme Fleury Perini,Nelson Hamerschlak

View Articletitled, Fine and Gray or Cox model?

Supplemental data

ERRATUM

Campbell BA, Dobos G, Haider Z, et al. International study of treatment efficacy in SS shows superiority of combination therapy and heterogeneity of treatment strategies. Blood Adv. 2023;7(21):6639-6647.

View Articletitled, Campbell BA, Dobos G, Haider Z, et al. International study of treatment efficacy in SS shows superiority of combination therapy and heterogeneity of treatment strategies. Blood Adv. 2023;7(21):6639-6647.

Supplemental data

Cover Image

Cover Image

Brain MRI of a patient with preexisting white matter disease who developed grade 3 immune effector cell–associated neurotoxicity syndrome (ICANS) following chimeric antigen receptor T-cell infusion. The white matter T2 hyperintensity shows progression from baseline (left column) to postinfusion during acute severe ICANS (right column). MRI images represent T2 FLAIR sequences, axial sections. See the article by Nie et al.

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Issue Archive

Table of Contents

INSIDE BLOOD ADVANCES

CLINICAL TRIALS AND OBSERVATIONS

Development of an umbilical cord blood transplantation–specific nonrelapse mortality risk assessment score Clinical Trials & Observations

von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease Clinical Trials & Observations

A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial Clinical Trials & Observations

Allogeneic hematopoietic cell transplantation for adult metachromatic leukodystrophy: a case series Clinical Trials & Observations

GENE THERAPY

HEALTH SERVICES AND OUTCOMES

IMMUNOBIOLOGY AND IMMUNOTHERAPY

LYMPHOID NEOPLASIA

IELSG30 phase 2 trial: intravenous and intrathecal CNS prophylaxis in primary testicular diffuse large B-cell lymphoma Clinical Trials & Observations

MYELOID NEOPLASIA

Phase 2 study of add-on parsaclisib for patients with myelofibrosis and suboptimal response to ruxolitinib: final results Clinical Trials & Observations

PLATELETS AND THROMBOPOIESIS

Prednisone vs high-dose dexamethasone in newly diagnosed adult primary immune thrombocytopenia: a randomized trial Clinical Trials & Observations

RED CELLS, IRON, AND ERYTHROPOIESIS

THROMBOSIS AND HEMOSTASIS

TRANSFUSION MEDICINE

TRANSPLANTATION

A multicenter study of posttransplantation low-dose inotuzumab ozogamicin to prevent relapse of acute lymphoblastic leukemia Clinical Trials & Observations

Liver abnormalities are frequent and persistent in patients with Fanconi anemia Clinical Trials & Observations

RESEARCH LETTERS

Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study Clinical Trials & Observations

Longitudinal plasma proteomics in CAR T–cell therapy patients implicates neutrophils and NETosis in the genesis of CRS Clinical Trials & Observations

Impact of imaging frequency on progression-free survival in Alliance trials enrolling patients with follicular lymphoma Clinical Trials & Observations

Large B-cell lymphoma-IRF4+ in children and young people: time to reduce chemotherapy in a rare malignant mature B-cell neoplasm? Clinical Trials & Observations

COMMENTARY

ERRATUM

Cover Image

Email alerts

ASH Publications

American Society of Hematology

This Feature Is Available To Subscribers Only

Development of an umbilical cord blood transplantation–specific nonrelapse mortality risk assessment score
Clinical Trials & Observations

von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease
Clinical Trials & Observations

A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial
Clinical Trials & Observations

Allogeneic hematopoietic cell transplantation for adult metachromatic leukodystrophy: a case series
Clinical Trials & Observations

IELSG30 phase 2 trial: intravenous and intrathecal CNS prophylaxis in primary testicular diffuse large B-cell lymphoma
Clinical Trials & Observations

Phase 2 study of add-on parsaclisib for patients with myelofibrosis and suboptimal response to ruxolitinib: final results
Clinical Trials & Observations

Prednisone vs high-dose dexamethasone in newly diagnosed adult primary immune thrombocytopenia: a randomized trial
Clinical Trials & Observations

A multicenter study of posttransplantation low-dose inotuzumab ozogamicin to prevent relapse of acute lymphoblastic leukemia
Clinical Trials & Observations

Liver abnormalities are frequent and persistent in patients with Fanconi anemia
Clinical Trials & Observations

Long-term efficacy and safety of romiplostim in refractory aplastic anemia: follow-up of a phase 2/3 study
Clinical Trials & Observations

Longitudinal plasma proteomics in CAR T–cell therapy patients implicates neutrophils and NETosis in the genesis of CRS
Clinical Trials & Observations

Impact of imaging frequency on progression-free survival in Alliance trials enrolling patients with follicular lymphoma
Clinical Trials & Observations

Large B-cell lymphoma-IRF4+ in children and young people: time to reduce chemotherapy in a rare malignant mature B-cell neoplasm?
Clinical Trials & Observations