Patricia L Musolino1, Troy C Lund2, Jessica Pan1, Maria L Escolar3, Asif M Paker1, Christine N Duncan4, Florian S Eichler1. 1. Department of Neurology, Massachusetts General Hospital, Boston, Massachusetts, United States. 2. Division of Pediatric Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, United States. 3. Department of Pediatrics, University of Pittsburgh, One Children's Hospital Drive, Pittsburgh, Pennsylvania, United States. 4. Dana-Farber Cancer Institute, Boston Children's Hospital, Boston, Massachusetts, United States.
Abstract
OBJECTIVE: The objective of this study is to systematically review the literature on worldwide numbers of leukodystrophy patients undergoing hematopoietic stem cell transplantation (HSCT) as well as the safety and efficacy of the procedure in this patient population. MATERIALS AND METHODS: A PubMed and EMBASE search up to June 2012 was conducted with a manual search of references from relevant articles. Selected studies were evaluated using internationally accepted criteria. The effect estimates of HSCT upon survival in early-stage disease versus late-stage disease were compared. RESULTS: One hundred and fifty-two studies qualified for inclusion and reported on a total of 689 patients. Study quality ranged from poor to good; no study was rated excellent. Small sample sizes limited most studies. Meta-analysis in a subset of larger studies indicates that transplantation in earlier stages of disease fairs better than in the late stages. Beyond survival, little longitudinal data on functional outcome is reported and neurological outcome is sparse. CONCLUSION: Further studies are needed to determine the neurological outcome following HSCT in the leukodystrophies. HSCT in the early stages of cerebral disease is still recommended for select leukodystrophies. Georg Thieme Verlag KG Stuttgart · New York.
OBJECTIVE: The objective of this study is to systematically review the literature on worldwide numbers of leukodystrophypatients undergoing hematopoietic stem cell transplantation (HSCT) as well as the safety and efficacy of the procedure in this patient population. MATERIALS AND METHODS: A PubMed and EMBASE search up to June 2012 was conducted with a manual search of references from relevant articles. Selected studies were evaluated using internationally accepted criteria. The effect estimates of HSCT upon survival in early-stage disease versus late-stage disease were compared. RESULTS: One hundred and fifty-two studies qualified for inclusion and reported on a total of 689 patients. Study quality ranged from poor to good; no study was rated excellent. Small sample sizes limited most studies. Meta-analysis in a subset of larger studies indicates that transplantation in earlier stages of disease fairs better than in the late stages. Beyond survival, little longitudinal data on functional outcome is reported and neurological outcome is sparse. CONCLUSION: Further studies are needed to determine the neurological outcome following HSCT in the leukodystrophies. HSCT in the early stages of cerebral disease is still recommended for select leukodystrophies. Georg Thieme Verlag KG Stuttgart · New York.
Authors: Maria L Escolar; Michele D Poe; James M Provenzale; Karen C Richards; June Allison; Susan Wood; David A Wenger; Daniel Pietryga; Donna Wall; Martin Champagne; Richard Morse; William Krivit; Joanne Kurtzberg Journal: N Engl J Med Date: 2005-05-19 Impact factor: 91.245
Authors: Paul L Martin; Shelly L Carter; Nancy A Kernan; Indira Sahdev; Donna Wall; Daniel Pietryga; John E Wagner; Joanne Kurtzberg Journal: Biol Blood Marrow Transplant Date: 2006-02 Impact factor: 5.742
Authors: E Shapiro; W Krivit; L Lockman; I Jambaqué; C Peters; M Cowan; R Harris; S Blanche; P Bordigoni; D Loes; R Ziegler; M Crittenden; D Ris; B Berg; C Cox; H Moser; A Fischer; P Aubourg Journal: Lancet Date: 2000-08-26 Impact factor: 79.321
Authors: Charles Peters; Lawrence R Charnas; Ye Tan; Richard S Ziegler; Elsa G Shapiro; Todd DeFor; Satkiran S Grewal; Paul J Orchard; Susan L Abel; Anne I Goldman; Norma K C Ramsay; Kathryn E Dusenbery; Daniel J Loes; Lawrence A Lockman; Shunichi Kato; Patrick R Aubourg; Hugo W Moser; William Krivit Journal: Blood Date: 2004-04-08 Impact factor: 22.113
Authors: Holly R Martin; Michele D Poe; James M Provenzale; Joanne Kurtzberg; Adam Mendizabal; Maria L Escolar Journal: Biol Blood Marrow Transplant Date: 2013-01-22 Impact factor: 5.742
Authors: Donald Beam; Michele D Poe; James M Provenzale; Paul Szabolcs; Paul L Martin; Vinod Prasad; Suhag Parikh; Tim Driscoll; Srini Mukundan; Joanne Kurtzberg; Maria L Escolar Journal: Biol Blood Marrow Transplant Date: 2007-04-06 Impact factor: 5.742
Authors: Thomas J Langan; Amy L Barcykowski; Jonathan Dare; Erin C Pannullo; Leah Muscarella; Randy L Carter Journal: J Neurosci Res Date: 2016-11 Impact factor: 4.164
Authors: Michael S Marshall; Yazan Issa; Benas Jakubauskas; Monika Stoskute; Vince Elackattu; Jeffrey N Marshall; Wil Bogue; Duc Nguyen; Zane Hauck; Emily Rue; Subha Karumuthil-Melethil; Violeta Zaric; Maarten Bosland; Richard B van Breemen; Maria I Givogri; Steven J Gray; Stephen J Crocker; Ernesto R Bongarzone Journal: Mol Ther Date: 2018-01-17 Impact factor: 11.454
Authors: Brigitte T A van den Broek; Kristin Page; Annalisa Paviglianiti; Janna Hol; Heather Allewelt; Fernanda Volt; Gerard Michel; Miguel Angel Diaz; Victoria Bordon; Tracey O'Brien; Peter J Shaw; Chantal Kenzey; Amal Al-Seraihy; Peter M van Hasselt; Andrew R Gennery; Eliane Gluckman; Vanderson Rocha; Annalisa Ruggeri; Joanne Kurtzberg; Jaap Jan Boelens Journal: Blood Adv Date: 2018-01-04
Authors: Gregory J Heller; Michael S Marshall; Yazan Issa; Jeffrey N Marshall; Duc Nguyen; Emily Rue; Koralege C Pathmasiri; Miriam S Domowicz; Richard B van Breemen; Leon M Tai; Stephanie M Cologna; Stephen J Crocker; Maria I Givogri; Mark S Sands; Ernesto R Bongarzone Journal: Mol Ther Date: 2021-01-26 Impact factor: 11.454
Authors: Thomas J Langan; Amy Barczykowski; Kabir Jalal; Laura Sherwood; Heather Allewelt; Joanne Kurtzberg; Randy L Carter Journal: JIMD Rep Date: 2019-04-11
Authors: Paul J Orchard; Todd W Markowski; LeeAnn Higgins; Gerald V Raymond; David R Nascene; Weston P Miller; Elizabeth I Pierpont; Troy C Lund Journal: Sci Rep Date: 2019-05-27 Impact factor: 4.379