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Journal Articles

In ALD, I feel the need for speed

Sandhya Kharbanda, Christopher C. Dvorak
Journal: Blood
Blood (2019) 133 (12): 1275–1277.
https://doi.org/10.1182/blood-2019-02-897629
Published: 2019
Journal Articles

Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy

Brief Report
Paul J. Orchard, David R. Nascene, Weston P. Miller, Ashish Gupta, Dan Kenney-Jung, Troy C. Lund
Journal: Blood
Blood (2019) 133 (12): 1378–1381.
https://doi.org/10.1182/blood-2018-11-887240
Published: 2019
Includes: Supplemental data
Journal Articles

Preliminary Phase 2 Results Demonstrate Engraftment with Minimal Neutropenia with MGTA-456, a CD34 + Expanded Cord Blood (CB) Product in Patients Transplanted for Inherited Metabolic Disorders (IMD)

Paul Orchard, MD, Glen D. Raffel, MD, Carolyn H Condon, MPH, Catherine A Monaghan, Demetra Vernet, Suzanne Tracey Sheirr, Jennifer Braun, RN BSN, Ryan Shanley, M.S, Troy C Lund, MD MS, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD, John C Davis, MD MPH, John E. Wagner, MD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3467.
https://doi.org/10.1182/blood-2018-99-115102
Published: 2018
Journal Articles

Reduced Toxicity Conditioning Is Better Tolerated but Has Higher Graft Failure Compared to Myeloablative Conditioning in Children with Inherited Metabolic Disorders

Michael Downey, BA, Ashish O. Gupta, MBBS, MPH, Ryan Shanley, M.S, Weston P. Miller, MD, Paul Orchard, MD, Angela R. Smith, MD MS
Journal: Blood
Blood (2018) 132 (Supplement 1): 3352.
https://doi.org/10.1182/blood-2018-99-117573
Published: 2018
Journal Articles

Repair of the Blood Brain Barrier and Neutrophil Recovery Following HSCT in Cerebral Adrenoleukodystrophy

Troy C Lund, MDMS, PhD, Weston P. Miller, MD, Dave Nascene, MD, Paul Orchard, MD, Ashish O. Gupta, MBBS, MPH
Journal: Blood
Blood (2018) 132 (Supplement 1): 4628.
https://doi.org/10.1182/blood-2018-99-109801
Published: 2018
Journal Articles

Two Cases Report of Haploid Hematopoietic Stem Cell Transplantation Treatment of Adrenal Leukodystrophy

Libai Chen, MD, Jianyun Wen, MD, Yuelin He, MD, Xiaoqin Feng, MDPhD, Chunfu Li, MD, Xuedong Wu, MD
Journal: Blood
Blood (2016) 128 (22): 5878.
https://doi.org/10.1182/blood.V128.22.5878.5878
Published: 2016
Journal Articles

High-Exposure, Targeted Daily Busulfan and Fludarabine-Based Conditioning for Children Undergoing Hematopoietic Stem Cell Transplantation for Inherited Metabolic Disorders: Outcomes at a Single Center

Weston P Miller, Melisa Kay Stricherz, PharmD, Cathryn Jennissen, PharmD, Nicole Sando, Jennifer Danielson, Troy Lund, Paul J Orchard
Journal: Blood
Blood (2016) 128 (22): 2191.
https://doi.org/10.1182/blood.V128.22.2191.2191
Published: 2016
Journal Articles

Haploidentical Stem Cell Transplantation for Rare Pediatric Diseases at Peking University People's Hospital

Yao Chen, MD, Huan Chen, MD, Lan-Ping Xu, Yu-Hong Chen, Jing-Zhi Wang, Feng-Rong Wang, Xiao-Hui Zhang, Kai-Yan Liu, Xiao-Jun Huang
Journal: Blood
Blood (2016) 128 (22): 5848.
https://doi.org/10.1182/blood.V128.22.5848.5848
Published: 2016
Journal Articles

A Pilot Trial of Unmatched Human Placenta-Derived Stem Cells (HPDSCs) in Conjunction with Unrelated Cord Blood Transplantation (UCBT) in Children and Young Adults with Malignant and Non-Malignant Disease (IND 14949)

Allyson M. Flower, MD, Michelle Nash, MD, Suzan Minzer, RN, MSN, FNP, Lauren Harrison, RN, MSN, Michael A Pulsipher, MD, Qiuhu Shi, PhD, Debra Abrams, MGC, CGC, MLIS, Roger H. Giller, MD, Erin Morris, RN, Olga Militano, PharmD, Janet Ayello, MS, Mildred Semidei-Pomales, Sandra Fabricatore, MSN, RN, PNP, Xiaokui Zhang, Jodi Gurney, Carmella van de Ven, MA, Cori M. Abikoff, MD, Jessica C. Hochberg, MD, Lee Ann Baxter-Lowe, PhD, Mitchell S. Cairo, MD
Journal: Blood
Blood (2016) 128 (22): 4671.
https://doi.org/10.1182/blood.V128.22.4671.4671
Published: 2016
Journal Articles

Neurocognitive Trajectory of Patients with Childhood Cerebral Adrenoleukodystrophy Who Received Allogeneic Hematopoietic Cell Transplantation at an Early Stage of Cerebral Disease

Elizabeth Pierpont, PhD, Julie Eisengart, PhD, Richard Ziegler, PhD, Gerald Raymond, Paul J Orchard, Troy Lund, Weston P Miller
Journal: Blood
Blood (2016) 128 (22): 4682.
https://doi.org/10.1182/blood.V128.22.4682.4682
Published: 2016
Journal Articles

Defibrotide for Prophylaxis of Hepatic Veno-Occlusive Disease in Pediatric Hematopoietic Stem Cell Transplantation: Subanalysis Data from an Open-Label, Phase III, Randomized Trial

Selim Corbacioglu, MD, Ansgar S. Schulz, MD, Petr Sedlacek, MD PhD, Bernd Gruhn, MD, Simone Cesaro, MD, Peter Bader, MD
Journal: Blood
Blood (2015) 126 (23): 4310.
https://doi.org/10.1182/blood.V126.23.4310.4310
Published: 2015
Journal Articles

Patient and Parental Perspectives on Long-Term Adaptive, Behavioral and Functional Outcomes Following Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders

Rachel Phelan, MD MPH, Paul J Orchard, MD, Margaret Semrud-Clikeman, PhD, Nicholas Smiley, Weston P Miller, IV, MD
Journal: Blood
Blood (2015) 126 (23): 4351.
https://doi.org/10.1182/blood.V126.23.4351.4351
Published: 2015
Journal Articles

Excellent Outcomes in Children and Young Adults Using Reduced-Intensity Conditioning for Patients with Inborn Errors of Immunity, Hematopoiesis, and Metabolism with Single-Unit Cord Blood or Bone Marrow

Mark Vander Lugt, MD, Hey Jin Chong, MD PhD, Xiaohua Chen, MDPhD, Randy M. Windreich, MD, Rakesh Goyal, MD MRCP, Maria Escolar, MD, Paul Szabolcs, MD
Journal: Blood
Blood (2015) 126 (23): 4329.
https://doi.org/10.1182/blood.V126.23.4329.4329
Published: 2015
Journal Articles

Outcomes after Donor Lymphocyte Infusion for Insufficient Donor Chimerism Following Hematopoietic Cell Transplantation for Non-Malignant Disorders

Carter Biewen, BA, Angela R Smith, MD MS, Jakub Tolar, MD, PhD, Weston P Miller, IV, MD
Journal: Blood
Blood (2015) 126 (23): 1969.
https://doi.org/10.1182/blood.V126.23.1969.1969
Published: 2015
Journal Articles

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Uimook Choi, PhD, Narda Theobald, Throm E Robert, PhD, John Gray, PhD, David J. Rawlings, MD, Brian P. Sorrentino, MD, Harry Malech, Suk See De Ravin, MD
Journal: Blood
Blood (2015) 126 (23): 2036.
https://doi.org/10.1182/blood.V126.23.2036.2036
Published: 2015
Journal Articles

Risk Factors for Graft Failure with Busulfan/Fludarabine-Based Conditioning in Children Undergoing Allogeneic Hematopoietic Cell Transplantation for Nonmalignant Disorders

Benjamin Huang, MD, Christopher C. Dvorak, MD, Justin Wahlstrom, MD, Jimmy Hwang, PhD, Janel R Long-Boyle, PharmD, PhD, Morton Jerome Cowan, MD, Biljana N. Horn, MD
Journal: Blood
Blood (2014) 124 (21): 1155.
https://doi.org/10.1182/blood.V124.21.1155.1155
Published: 2014
Journal Articles

Allogeneic Stem Cell Transplantation For Patients With Adrenoleukodystrophy. Nationwide Retrospective Study In Japan

Koji Kato, MD, PhD, Hiromasa Yabe, MD, Shunichi Kato, Souichi Adachi, MD, PhD, Yoshiko Hashii, MD, PhD, Keisei Kawa, MD, PhD, Yoshiko Atsuta, MD
Journal: Blood
Blood (2013) 122 (21): 2148.
https://doi.org/10.1182/blood.V122.21.2148.2148
Published: 2013
Journal Articles

Outcomes Following Allogeneic Hematopoietic Stem Cell Transplantation For Metachromatic Leukodystrophy

Alexander A Boucher, MD, Gerald V Raymond, MD, Ryan Shanley, Paul J Orchard, MD, Weston P Miller, IV, MD
Journal: Blood
Blood (2013) 122 (21): 714.
https://doi.org/10.1182/blood.V122.21.714.714
Published: 2013
Journal Articles

Haploidentical Hematopoietic Stem Cell Transplantation: A New Transplantation Modality Implemented In a Recent Accredited FACT Institution In Latin America

Iracema Esteteves, PhD, Fabio PS Santos, PhD, Morgani Rodrigues, PhD, Jairo Jose, Nascimento Sobrinho, Ricardo Helman, MD, Guilherme Fleury Perini, MD, Reijane Alves de Assis, Jr., MD, Andreza Alice Feitosa Ribeiro, PhD, Jose Mauro Kutner, MD, PhD, Araci M. Sakashita, MD, Margareth Torres, Physician, Leonardo Raul Morelli, PhD, Claudia Mac-Donald Bley do Nascimento, MD, Alessandro de Moura Almeida, PhD, Joyce Esteves Hippolito, Andrea Tiemi Kondo, MD, Juliana Folloni Fernandes, PhD, Fabio Rodrigues Kerbauy, PhD, Nelson Hamerschlak, MD, PhD
Journal: Blood
Blood (2013) 122 (21): 5596.
https://doi.org/10.1182/blood.V122.21.5596.5596
Published: 2013
Journal Articles

Outcomes Of Allogeneic Cord Blood Transplantation For Leukodystrophies; A Joint Study of Eurocord and “Inborn Errors WP-EBMT”

Hol Janna, Annalisa Ruggeri, MD, Vanderson Rocha, MD, Gerard Michel, Mouhab Ayas, MD, Tracey O'Brien, Gergely Krivan, Bordon Victoria, Rik Schots, MD, PhD, Rovelli Attilio, Elorza Izaskun, Jose M Moraleda, Peter J. Shaw, MBBS, MA, Marleen Renard, Karl-Walter Sykora, MD, Reuven Or, MD, Tsila Zuckerman, MD, Manuel Abecasis, Bredius Robbert, M. Akif Yesilipek, Miguel Angel Diaz, MD, PhD, Gennery Andy, Eliane Gluckman, MD, Jaap Jan Boelens, MD PhD
Journal: Blood
Blood (2013) 122 (21): 2101.
https://doi.org/10.1182/blood.V122.21.2101.2101
Published: 2013