Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency

Stephanie van Straaten; Marc Bierings; Paola Bianchi; Kensuke Akiyoshi; Hitoshi Kanno; Isabel Badell Serra; Jing Chen; Xiaohang Huang; Eduard van Beers; Supachai Ekwattanakit; Tayfun Güngör; Wijnanda Adriana Kors; Frans Smiers; Reinier Raymakers; Lucrecia Yanez; Julian Sevilla; Wouter van Solinge; Jose Carlos Segovia; Richard van Wijk

doi:10.3324/haematol.2017.177857

Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency

Haematologica. 2018 Feb;103(2):e82-e86. doi: 10.3324/haematol.2017.177857. Epub 2017 Dec 14.

Authors

Stephanie van Straaten^{1

2}, Marc Bierings³, Paola Bianchi⁴, Kensuke Akiyoshi⁵, Hitoshi Kanno⁶, Isabel Badell Serra⁷, Jing Chen⁸, Xiaohang Huang⁸, Eduard van Beers⁹, Supachai Ekwattanakit¹⁰, Tayfun Güngör¹¹, Wijnanda Adriana Kors¹², Frans Smiers¹³, Reinier Raymakers¹⁴, Lucrecia Yanez¹⁵, Julian Sevilla¹⁶, Wouter van Solinge¹, Jose Carlos Segovia^{17

18}, Richard van Wijk¹⁹

Affiliations

¹ Department of Clinical Chemistry and Haematology, University Medical Center Utrecht, Utrecht University, the Netherlands.
² Van CreveldKliniek, University Medical Center Utrecht, Utrecht University, the Netherlands.
³ Pediatric Blood and Marrow Transplantation Program, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, the Netherlands.
⁴ Hematology Unit, Anemia Physiopathology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
⁵ Department of Pediatrics and Child Neurology, Oita University Faculty of Medicine, Hasama, Yufu, Japan.
⁶ Department of Transfusion Medicine and Cell Processing, Faculty of Medicine, Tokyo Women's Medical University, Japan.
⁷ Directora Unidad Pediátrica de Trasplante Hematopoyético, Hospital Santa Creu i Sant Pau, Barcelona, Spain.
⁸ Department of Hematology and Oncology, Shanghai Children's Medical Center. Shanghai Jiao Tong University School of Medicine, China.
⁹ Van Creveldkliniek, Department of Internal Medicine, University Medical Center Utrecht, Utrecht University, the Netherlands.
¹⁰ Department of Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
¹¹ Division of Stem Cell Transplantation, University Children's Hospital, Zurich, Switzerland.
¹² Department of Pediatric Oncology, VU University Medical Center, Amsterdam, the Netherlands.
¹³ Department of Pediatric Haematology, Leiden University Hospital, the Netherlands.
¹⁴ Department of Internal Medicine, University Medical Center Utrecht, Utrecht University, the Netherlands.
¹⁵ Servicio de Hematología, Hospital Universitario Marqués de Valdecilla, Santander, Spain.
¹⁶ Servicio Hemato-Oncología Pediatrica, Hospital Infantil Universitario Niño Jesús, Madrid, Spain.
¹⁷ Differentiation and Cytometry Unit, Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigaciones Biomédicas en Red de Enfermedades Raras (CIBERER), Madrid, Spain.
¹⁸ Advance Therapies Mixed Unit, Instituto de Investigación Sanitaria-Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain.
¹⁹ Department of Clinical Chemistry and Haematology, University Medical Center Utrecht, Utrecht University, the Netherlands [email protected].

No abstract available

MeSH terms

Adolescent
Adult
Anemia, Hemolytic / etiology
Anemia, Hemolytic, Congenital Nonspherocytic / complications
Anemia, Hemolytic, Congenital Nonspherocytic / epidemiology
Anemia, Hemolytic, Congenital Nonspherocytic / mortality
Anemia, Hemolytic, Congenital Nonspherocytic / therapy*
Child
Child, Preschool
Erythrocyte Transfusion
Female
Follow-Up Studies
Graft vs Host Disease / etiology
Hematopoietic Stem Cell Transplantation / adverse effects
Hematopoietic Stem Cell Transplantation / methods*
Hematopoietic Stem Cell Transplantation / mortality
Humans
Male
Pyruvate Kinase / deficiency*
Pyruvate Metabolism, Inborn Errors / complications
Pyruvate Metabolism, Inborn Errors / epidemiology
Pyruvate Metabolism, Inborn Errors / mortality
Pyruvate Metabolism, Inborn Errors / therapy*
Retrospective Studies
Survival Analysis
Young Adult

Substances

Pyruvate Kinase

Supplementary concepts

Pyruvate Kinase Deficiency of Red Cells