Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use

Autor: Gilbert MM, Grimes AB, Kim TO, Despotovic JM

Publikováno v: Patient Preference and Adherence, Vol Volume 14, Pp 1237-1250 (2020)

Megan M Gilbert, Amanda B Grimes, Taylor Olmsted Kim, Jenny M Despotovic Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, USACorrespondence: Megan M GilbertDepartment of Pediatrics, Section of Hematol

Externí odkaz: https://doaj.org/article/08e2de8c2800452e87b96732423e737a

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.

Autor: Labrosse R; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Immune Deficiency-Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.; Division of Allergy and Immunology, Department of Pediatrics, CHU Sainte-Justine, University of Montreal, Montreal, QC, Canada., Chu JI; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Division of Pediatric Allergy, Immunology and Bone Marrow Transplantation, Benioff Children's Hospital, University of California San Francisco, San Francisco, CA., Armant MA; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Everett JK; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Pellin D; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Kareddy N; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Frelinger AL; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Henderson LA; Division of Immunology, Boston Children's Hospital, Boston, MA., O'Connell AE; Division of Newborn Medicine, Boston Children's Hospital, Boston, MA., Biswas A; Division of Gastroenterology, Hepatology and Nutrition, Boston Children's Hospital, Harvard Medical School, Boston, MA., Coenen-van der Spek J; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Miggelbrink A; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Fiorini C; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Adhikari H; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Berry CC; Division of Biostatistics and Bioinformatics, Herbert Wertheim School of Public Health, UC San Diego, La Jolla, CA., Cantu VA; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Fong J; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Jaroslavsky J; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Karadeniz DF; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Li QZ; Department of Immunology, Microarray and Immune Phenotyping Core Facility, University of Texas Southwestern Medical Center, Dallas, TX., Reddy S; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Roche AM; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Zhu C; Department of Immunology, Microarray and Immune Phenotyping Core Facility, University of Texas Southwestern Medical Center, Dallas, TX., Whangbo JS; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Dansereau C; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Mackinnon B; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Morris E; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., Koo SM; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA., London WB; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Baris S; Department of Pediatrics, Division of Allergy and Immunology, Marmara University, Istanbul, Turkey.; The Işıl Berat Barlan Center for Translational Medicine, Marmara University, Istanbul, Turkey., Ozen A; Department of Pediatrics, Division of Allergy and Immunology, Marmara University, Istanbul, Turkey.; The Işıl Berat Barlan Center for Translational Medicine, Marmara University, Istanbul, Turkey., Karakoc-Aydiner E; Department of Pediatrics, Division of Allergy and Immunology, Marmara University, Istanbul, Turkey.; The Işıl Berat Barlan Center for Translational Medicine, Marmara University, Istanbul, Turkey., Despotovic JM; Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, TX., Forbes Satter LR; Department of Pediatrics, Baylor College of Medicine, Texas Children's Hospital, Houston, TX., Saitoh A; Department of Pediatrics, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan., Aizawa Y; Department of Pediatrics, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan., King A; Hospital Luis Calvo Mackenna, Clínica Alemana De Santiago Universidad del Desarrollo, Santiago, Chile., Nguyen MAT; Department of Pediatrics, University of Medicine and Pharmacy at Ho Chi Minh City, Ho Chi Minh City, Vietnam., Vu VDU; Department of Pediatrics, University of Medicine and Pharmacy at Ho Chi Minh City, Ho Chi Minh City, Vietnam., Snapper SB; Division of Gastroenterology, Hepatology and Nutrition, Boston Children's Hospital, Harvard Medical School, Boston, MA., Galy A; Genethon, Évry, France.; University of Paris-Saclay, University of Évry, INSERM, Genethon, Integrare Research Unit UMR_S951, Évry, France., Notarangelo LD; Division of Immunology, Boston Children's Hospital, Boston, MA.; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD., Bushman FD; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Williams DA; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA., Pai SY; Division of Pediatric Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.; Immune Deficiency-Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD.

Publikováno v: Blood [Blood] 2023 Oct 12; Vol. 142 (15), pp. 1281-1296.

Proceedings of the immune thrombocytopenia summit: new concepts in mechanisms, diagnosis, and management.

Autor: Mahamad S; Department of Medicine, McMaster University, Hamilton, Ontario, Canada., Modi D; Department of Medicine, McMaster University, Hamilton, Ontario, Canada.; Department of Laboratory Medicine and Molecular Diagnostics, Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada., Al-Samkari H; Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA., Cuker A; Department of Medicine and Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Despotovic JM; Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA., Italiano JE; Department of Surgery, Vascular Biology Program, Boston Children's Hospital, Boston, Massachusetts, USA., Lambert MP; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Lee EJ; Hematology/Oncology Department, Weill Cornell Medical College, New York, New York, USA., Rondina MT; Department of Internal Medicine & The Molecular Medicine Program, University of Utah, Salt Lake City, Utah, USA.; Department of Internal Medicine, George E. Wahlen Department of Veterans Affairs Medical Center, Salt Lake City, Utah, USA.; University of Utah, Department of Pathology, Salt Lake City, Utah, USA., Sholzberg M; Department of Medicine, and Laboratory Medicine and Pathobiology, St. Michael's Hospital, Toronto, Ontario, Canada.; Li Ka Shing Knowledge Institute, Toronto, Ontario, Canada., Kruse C; Platelet Disorder Support Association, Cleveland, Ohio, USA., Larché M; Division of Clinical Immunology & Allergy, and Respirology, Department of Medicine, McMaster University, Hamilton, Ontario, Canada.; Firestone Institute for Respiratory Health, McMaster University, Hamilton, Ontario, Canada., Nazy I; Division of Hematology and Thromboembolism, Department of Medicine, McMaster University, Hamilton, Ontario, Canada., Miller MS; Department of Biochemistry and Biomedical Sciences, Michael G. DeGroote Institute for Infectious Disease Research, McMaster Immunology Research Centre, McMaster University, Hamilton, Ontario, Canada., Arnold DM; Department of Medicine, McMaster University, Hamilton, Ontario, Canada.

Publikováno v: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2023 Feb 27; Vol. 7 (2), pp. 100097. Date of Electronic Publication: 2023 Feb 27 (Print Publication: 2023).

Diagnosis, monitoring, and management of pyruvate kinase deficiency in children.

Autor: Johnson S; Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA., Grace RF; Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, Massachusetts, USA.; Harvard Medical School, Boston, Massachusetts, USA., Despotovic JM; Department of Pediatrics, Division of Hematology/Oncology, Baylor College of Medicine, Houston, Texas, USA.; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, Texas, USA.

Publikováno v: Pediatric blood & cancer [Pediatr Blood Cancer] 2022 Aug; Vol. 69 (8), pp. e29696. Date of Electronic Publication: 2022 Apr 22.

Molecular Diagnosis Is Vital to the Accurate Classification and Management of Thrombotic Thrombocytopenic Purpura in Children.

Autor: Karsenty CL; Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, United States.; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, TX, United States., Kirk SE; Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, United States.; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, TX, United States., Helber HL; Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, United States.; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, TX, United States., Esquilin JM; Methodist Children's Hospital, San Antonio, TX, United States.; Methodist Physicians Pediatric Specialists of Texas, San Antonio, TX, United States., Despotovic JM; Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, United States.; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, TX, United States., Grimes AB; Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, United States.; Texas Children's Cancer and Hematology Centers, Texas Children's Hospital, Houston, TX, United States.

Publikováno v: Frontiers in immunology [Front Immunol] 2022 Apr 11; Vol. 13, pp. 836960. Date of Electronic Publication: 2022 Apr 11 (Print Publication: 2022).