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Journal Articles

Ruxolitinib for pediatric patients with treatment-naïve and steroid-refractory acute graft-versus-host disease: the REACH4 study

Franco Locatelli, Hyoung Jin Kang, Bénédicte Bruno, Virginie Gandemer, Fanny Rialland, Maura Faraci, Yoshiyuki Takahashi, Katsuyoshi Koh, Henrique Bittencourt, Grace Cleary, Christine Rosko, Xuechan Li, Annie St Pierre, Anirudh Prahallad, Cristina Diaz-de-Heredia

Blood (2024) 144 (20): 2095–2106.

https://doi.org/10.1182/blood.2023022565

Published: 2024

Includes: Supplemental data

Journal Articles

Severe Congenital Neutropenia-Type 5: Impaired T Cell Proliferation, Aberrant Th1 Cytokine Production, Abnormal Megakaryocytes, and Impaired Platelet Granule Formation in a Patient with VPS45 Deficiency Caused By Uniparental Isodisomy

Masahiro Ueki, Shinsuke Hirabayashi, Daichi Sajiki, Masataka Hasegawa, Yukayo Terashita, Hiroki Ohata, Manabu Wakamatsu, Shunichiro Takezaki, Yuko Cho, Kazuhiko Nakabayashi, Hideki Muramatsu, Takahiro Yamada, Yoshiyuki Takahashi, Atsushi Manabe

Blood (2023) 142 (Supplement 1): 5382.

https://doi.org/10.1182/blood-2023-183059

Published: 2023

Journal Articles

Prevalence and Outcome of Vexas Syndrome in Unrelated Hematopoietic Stem Cell Transplantation

Blood (2023) 142 (Supplement 1): 1354.

https://doi.org/10.1182/blood-2023-190791

Published: 2023

Journal Articles

Diagnostic Utility of Proteogenomic Analysis for Inherited Bone Marrow Failure Syndrome

Manabu Wakamatsu, Hideki Muramatsu, Hironori Sato, Masaki Ishikawa, Daisuke Nakajima, Ryo Konno, Yusuke Kawashima, Motoharu Hamada, Yusuke Okuno, Osamu Ohara, Yoshiyuki Takahashi

Blood (2023) 142 (Supplement 1): 1363.

https://doi.org/10.1182/blood-2023-178288

Published: 2023

Journal Articles

Error-Corrected Next-Generation Sequencing Provides a Comprehensive Overview of the Subclonal Mutation Landscape and Its Prognostic Implications in Juvenile Myelomonocytic Leukemia

Daichi Sajiki, Manabu Wakamatsu, Hideki Muramatsu, Yusuke Tsumura, Daiki Yamashita, Ryo Maemura, Ayako Yamamori, Kotaro Narita, Shinsuke Kataoka, Atsushi Narita, Noriko Shimasaki, Nobuhiro Nishio, Yoshiyuki Takahashi

Blood (2023) 142 (Supplement 1): 3199.

https://doi.org/10.1182/blood-2023-189160

Published: 2023

Journal Articles

Ruxolitinib in Pediatric Patients with Treatment- Naïve or Steroid-Refractory Acute Graft-Versus-Host Disease: Primary Findings from the Phase I/II REACH4 Study

Franco Locatelli, Hyoung Jin Kang, Bénédicte Bruno, Virginie Gandemer, Fanny Rialland, Maura Faraci, Yoshiyuki Takahashi, Katsuyoshi Koh, Henrique Bittencourt, Grace Cleary, Christine Rosko, Pantelia Roussou, Annie St. Pierre, Anirudh Prahallad, Cristina Díaz-de-Heredia

Blood (2022) 140 (Supplement 1): 1376–1378.

https://doi.org/10.1182/blood-2022-155708

Published: 2022

Journal Articles

A Phase I Study of CD19 Chimeric Antigen Receptor-T Cells Generated By the PiggyBac Transposon Vector for Acute Lymphoblastic Leukemia

Nobuhiro Nishio, Ryo Hanajiri, Yuichi Ishikawa, Makoto Murata, Rieko Taniguchi, Motoharu Hamada, Eri Nishikawa, Nozomu Kawashima, Atsushi Narita, Hideki Muramatsu, Yoshiyuki Takahashi

Blood (2021) 138 (Supplement 1): 3831.

https://doi.org/10.1182/blood-2021-150469

Published: 2021

Journal Articles

CAR2.0 Therapy for the Management of Post-Transplantation Relapse of B-Cell Acute Lymphoblastic Leukemia

Blood (2020) 136 (Supplement 1): 7.

https://doi.org/10.1182/blood-2020-141531

Published: 2020

Journal Articles

Diagnostic Whole Exome Sequencing for 166 Patients with Inherited Bone Marrow Failure Syndrome

Blood (2020) 136 (Supplement 1): 9.

https://doi.org/10.1182/blood-2020-143241

Published: 2020

Journal Articles

DNA Methylation As a Biomarker of Outcome in JMML: An International Effort Towards Clinical Implementation

Julia Meyer, PhD, Maximillian Schönung, Christian Flotho, MD PhD, Adam B. Olshen, PhD, Mark Hartmann, PhD, Christoph Plass, PhD, Yusuke Okuno, MD PhD, Yoshiyuki Takahashi, MD PhD, Norihiro Murakami, MD PhD, Manabu Wakamatsu, MD, Mignon L. Loh, MD, Charlotte M. Niemeyer, MD, Hideki Muramatsu, MD PhD, Daniel B. Lipka, MD, Elliot Stieglitz, MD

Blood (2019) 134 (Supplement_1): 1693.

https://doi.org/10.1182/blood-2019-131279

Published: 2019

Journal Articles

Phase I Study of Cord Blood Transplantation with Intra-Bone Marrow Injection of Mesenchymal Stem Cells

Tatsunori Goto, MD, Makoto Murata, MD PhD, Tetsuya Nishida, MD PhD, Seitaro Terakura, MD PhD, Sonoko Kamoshita, MD, Yuichi Ishikawa, MD PhD, Yoko Ushijima, MD PhD, Yoshiya Adachi, MD PhD, Katsuyoshi Kato, PhD, Akihiro Hirakawa, PhD, Satoshi Nishiwaki, MD PhD, Nobuhiro Nishio, MD PhD, Yoshiyuki Takahashi, MD PhD, Yoshihisa Kodera, MD, Tadashi Matsushita, Hitoshi Kiyoi, MD PhD

Blood (2019) 134 (Supplement_1): 2004.

https://doi.org/10.1182/blood-2019-122101

Published: 2019

Journal Articles

Utility of Newborn Screening for Severe Combined Immunodeficiency and X-Linked Agammaglobulinemia Using TREC and KREC Assays

Manabu Wakamatsu, MD, Hideki Muramatsu, MD PhD, Shinsuke Kataoka, MD, Yusuke Okuno, MD PhD, Sakai Yoshimi, Yoko Nakajima, MD PhD, Tetsuya Ito, MD PhD, Ikuya Tsuge, MD PhD, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD PhD

Blood (2019) 134 (Supplement_1): 3604.

https://doi.org/10.1182/blood-2019-126669

Published: 2019

Journal Articles

Genome-Wide Methylation Analysis Using the Digital Restriction Enzyme Analysis of Methylation for Stratification of Patients with Juvenile Myelomonocytic Leukemia

Hironobu Kitazawa, Hideki Muramatsu, MD PhD, Norihiro Murakami, MD PhD, Yusuke Okuno, MD PhD, Manabu Wakamatsu, MD, Taro Yoshida, MD, Masayuki Imaya, MD, Ayako Yamamori, MD, Shunsuke Miwata, MD, Kotaro Narita, MD, Motoharu Hamada, MD PhD, Daisuke Ichikawa, MD, Rieko Taniguchi, MD, Nozomu Kawashima, MD PhD, Eri Nishikawa, MD PhD, Atsushi Narita, Nobuhiro Nishio, MD PhD, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD PhD

Blood (2019) 134 (Supplement_1): 2973.

https://doi.org/10.1182/blood-2019-127792

Published: 2019

Journal Articles

Comprehensive Mutational Analysis of Juvenile Myelomonocytic Leukemia Using Whole-Genome Sequencing

Yusuke Okuno, MD PhD, Hideki Muramatsu, MD PhD, Norihiro Murakami, MD PhD, Nozomu Kawashima, MD, Manabu Wakamatsu, MD, Hironobu Kitazawa, Tomoo Ogi, PhD, Yoshiyuki Takahashi, MD PhD

Blood (2019) 134 (Supplement_1): 2974.

https://doi.org/10.1182/blood-2019-121681

Published: 2019

Journal Articles

High Natural Killer Cell Count at Diagnosis Predicts a Good Response to Immunosuppressive Therapy in Aplastic Anemia

Atsushi Narita, Hideki Muramatsu, MD PhD, Yinyan Xu, Motoharu Hamada, MD PhD, Daisuke Ichikawa, MD, Eri Nishikawa, MD PhD, Nozomu Kawashima, MD, Yusuke Okuno, MD PhD, Nobuhiro Nishio, MD PhD, Hirohito Yamazaki, MD PhD, Shinji Nakao, MD PhD, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD PhD

Blood (2019) 134 (Supplement_1): 2505.

https://doi.org/10.1182/blood-2019-124945

Published: 2019

Journal Articles

Detection of Subclonal SETBP1 and JAK3 Mutations in Patients with Juvenile Myelomonocytic Leukemia Using Droplet Digital PCR

Manabu Wakamatsu, MD, Hideki Muramatsu, MD PhD, Norihiro Murakami, MD PhD, Yusuke Okuno, MD PhD, Hironobu Kitazawa, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD PhD

Blood (2019) 134 (Supplement_1): 4213.

https://doi.org/10.1182/blood-2019-125354

Published: 2019

Journal Articles

Randomized Trial of Two Dosages of Rabbit Antithymocyte Globulin in Patients with Aplastic Anemia

Atsushi Narita, MD PhD, Xiaofan Zhu, PhD, Hideki Muramatsu, MD PhD, Xiaojuan Chen, Ye Guo, Wenyu Yang, Jingliao Zhang, MD, Fang Liu, Jun Ho Jang, MD PhD, Hoon Kook, MD PhD, Hawk Kim, MD PhD, Kensuke Usuki, MD PhD, Hirohito Yamazaki, MD PhD, Yoshiyuki Takahashi, MD, PhD, Shinji Nakao, MD PhD, Jong-Wook Lee, MD PhD, Seiji Kojima, MD PhD

Blood (2018) 132 (Supplement 1): 3873.

https://doi.org/10.1182/blood-2018-99-111323

Published: 2018

Journal Articles

Combination of TREC Measurement and Next-Generation Sequencing in Newborn Screening for Severe Combined Immunodeficiency: A Pilot Program in Japan

Hideki Muramatsu, MD PhD, Daiei Kojima, MD, Yusuke Okuno, MD PhD, Shinsuke Kataoka, MD, Yoko Nakajima, Tetsuya Ito, Ikuya Tsuge, Sakai Yoshimi, Tomoaki Kato, Seiji Kojima, MD PhD, Yoshiyuki Takahashi, MD, PhD

Blood (2018) 132 (Supplement 1): 3717.

https://doi.org/10.1182/blood-2018-99-118261

Published: 2018

Journal Articles

Piggybac CD19 CAR T Cells Eradicate CNS Leukemia By Direct Delivery into Cerebral Ventricle of Xenograft Mice Model

Kuniaki Tanaka, MD, Itaru Kato, MD, PhD, Miyuki Tanaka, MD, PhD, Daisuke Morita, MD, PhD, Yoshiyuki Takahashi, MD, PhD, Tatsutoshi Nakahata, MD, PhD, Katsutsugu Umeda, MD, PhD, Hidefumi Hiramatsu, MD, PhD, Souichi Adachi, MD PhD, Junko Takita, MD, PhD, Yozo Nakazawa, MD, PhD

Blood (2018) 132 (Supplement 1): 4028.

https://doi.org/10.1182/blood-2018-99-111733

Published: 2018

Journal Articles

Clinical and Genetic Characteristics of Patients with Shwachman-Diamond Syndrome in Japan

Kenichiro Watanabe, MD PhD, Hirokazu Kanegane, MD PhD, Takayuki Hamabata, MD, Kagehiro Kozuki, MD, Katsutsugu Umeda, MD, PhD, Hiroo Ueno, MD, Kenichi Yoshida, MD PhD, Asahito Hama, Yusuke Okuno, MD PhD, Hideki Muramatsu, MD PhD, Yoshiyuki Takahashi, MD, PhD, Daisuke Hasegawa, MD PhD, Atsushi Manabe, MD PhD, Akira Ohara, MD, Masafumi Ito, MD PhD, Seishi Ogawa, MD PhD, Seiji Kojima, MD PhD, Etsuro Ito, MD PhD

Blood (2018) 132 (Supplement 1): 3862.

https://doi.org/10.1182/blood-2018-99-114313

Published: 2018

NARROW

Ruxolitinib for pediatric patients with treatment-naïve and steroid-refractory acute graft-versus-host disease: the REACH4 study

Severe Congenital Neutropenia-Type 5: Impaired T Cell Proliferation, Aberrant Th1 Cytokine Production, Abnormal Megakaryocytes, and Impaired Platelet Granule Formation in a Patient with VPS45 Deficiency Caused By Uniparental Isodisomy

Prevalence and Outcome of Vexas Syndrome in Unrelated Hematopoietic Stem Cell Transplantation

Diagnostic Utility of Proteogenomic Analysis for Inherited Bone Marrow Failure Syndrome

Error-Corrected Next-Generation Sequencing Provides a Comprehensive Overview of the Subclonal Mutation Landscape and Its Prognostic Implications in Juvenile Myelomonocytic Leukemia

Ruxolitinib in Pediatric Patients with Treatment- Naïve or Steroid-Refractory Acute Graft-Versus-Host Disease: Primary Findings from the Phase I/II REACH4 Study

A Phase I Study of CD19 Chimeric Antigen Receptor-T Cells Generated By the PiggyBac Transposon Vector for Acute Lymphoblastic Leukemia

CAR2.0 Therapy for the Management of Post-Transplantation Relapse of B-Cell Acute Lymphoblastic Leukemia

Diagnostic Whole Exome Sequencing for 166 Patients with Inherited Bone Marrow Failure Syndrome

DNA Methylation As a Biomarker of Outcome in JMML: An International Effort Towards Clinical Implementation

Phase I Study of Cord Blood Transplantation with Intra-Bone Marrow Injection of Mesenchymal Stem Cells

Utility of Newborn Screening for Severe Combined Immunodeficiency and X-Linked Agammaglobulinemia Using TREC and KREC Assays

Genome-Wide Methylation Analysis Using the Digital Restriction Enzyme Analysis of Methylation for Stratification of Patients with Juvenile Myelomonocytic Leukemia

Comprehensive Mutational Analysis of Juvenile Myelomonocytic Leukemia Using Whole-Genome Sequencing

High Natural Killer Cell Count at Diagnosis Predicts a Good Response to Immunosuppressive Therapy in Aplastic Anemia

Detection of Subclonal SETBP1 and JAK3 Mutations in Patients with Juvenile Myelomonocytic Leukemia Using Droplet Digital PCR

Randomized Trial of Two Dosages of Rabbit Antithymocyte Globulin in Patients with Aplastic Anemia

Combination of TREC Measurement and Next-Generation Sequencing in Newborn Screening for Severe Combined Immunodeficiency: A Pilot Program in Japan

Piggybac CD19 CAR T Cells Eradicate CNS Leukemia By Direct Delivery into Cerebral Ventricle of Xenograft Mice Model

Clinical and Genetic Characteristics of Patients with Shwachman-Diamond Syndrome in Japan

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