Daniela Melis1, Giorgia Minopoli2, Francesca Balivo2, Paola Marcolongo3, Rossella Parini4, Sabrina Paci5, Carlo Dionisi-Vici6, Roberto Della Casa7, Angelo Benedetti3, Generoso Andria7, Giancarlo Parenti7,8. 1. Dipartimento di Pediatria, Università "Federico II", Naples, Italy. daniela.melis@unina.it. 2. Dipartimento di Pediatria, Università "Federico II", Naples, Italy. 3. Dipartimento di Biologia, Università degli Studi di Siena, Siena, Italy. 4. Ospedale S. Gerardo, Monza, Italy. 5. Ospedale S. Paolo, Milan, Italy. 6. Ospedale Pediatrico Bambino Gesù, Rome, Italy. 7. Dipartimento di Scienze Mediche Traslazionali, Sezione di Pediatria, Università degli Studi "Federico II", Naples, Italy. 8. Telethon Institute of Genetics and Medicine, Naples, Italy.
Abstract
BACKGROUND: It has been suggested, on a few GSD1b patients, that vitamin E improves neutrophil count and reduces frequency and severity of infections.The main objective of the present study was to investigate the efficacy of vitamin E on the neutropenia, neutrophil dysfunction and IBD in the entire Italian caseload of GSD1b patients. PATIENTS AND METHODS: Eighteen GSD1b patients, median age at the time of the study protocol 14.5 (range, 0.6-42 years), were enrolled from four Italian referral centres for metabolic diseases. For the evaluation of the efficacy of vitamin E, neutrophil count and function, frequency of infections needing hospitalization and inflammatory bowel activity were evaluated periodically all over one year before and during vitamin E therapy. RESULTS: Frequency (1.5 ± 0.1 vs. 6.0 ± 0.6, p = 0.003) and severity of infections (2.2 ± 0.2 vs. 3.7 ± 0.4, p = 0.003) were lower and mean value of neutrophil count (1,583 ± 668 vs. 941 ± 809, p = 0.03) higher during vitamin E supplementation. Neutrophil function results improved during vitamin supplementation. PCDAI showed a significant reduction in the inflammatory activity during vitamin E supplementation (9 ± 1.4 vs. 13 ± 1.2, p = 0.006). In seven patients G-CSF requirement decreased and the dose was reduced after the end of the study.In conclusion, our study demonstrated the efficacy of vitamin E supplementation. Vitamin E has evident advantages as compared to G-CSF, as it can be assumed orally, and it has not been associated with severe side effects.
BACKGROUND: It has been suggested, on a few GSD1bpatients, that vitamin E improves neutrophil count and reduces frequency and severity of infections.The main objective of the present study was to investigate the efficacy of vitamin E on the neutropenia, neutrophil dysfunction and IBD in the entire Italian caseload of GSD1bpatients. PATIENTS AND METHODS: Eighteen GSD1bpatients, median age at the time of the study protocol 14.5 (range, 0.6-42 years), were enrolled from four Italian referral centres for metabolic diseases. For the evaluation of the efficacy of vitamin E, neutrophil count and function, frequency of infections needing hospitalization and inflammatory bowel activity were evaluated periodically all over one year before and during vitamin E therapy. RESULTS: Frequency (1.5 ± 0.1 vs. 6.0 ± 0.6, p = 0.003) and severity of infections (2.2 ± 0.2 vs. 3.7 ± 0.4, p = 0.003) were lower and mean value of neutrophil count (1,583 ± 668 vs. 941 ± 809, p = 0.03) higher during vitamin E supplementation. Neutrophil function results improved during vitamin supplementation. PCDAI showed a significant reduction in the inflammatory activity during vitamin E supplementation (9 ± 1.4 vs. 13 ± 1.2, p = 0.006). In seven patientsG-CSF requirement decreased and the dose was reduced after the end of the study.In conclusion, our study demonstrated the efficacy of vitamin E supplementation. Vitamin E has evident advantages as compared to G-CSF, as it can be assumed orally, and it has not been associated with severe side effects.
Authors: Douglas L Seidner; Bret A Lashner; Aaron Brzezinski; Phillip L C Banks; John Goldblum; Claudio Fiocchi; Jeffry Katz; Gary R Lichtenstein; Peter A Anton; Lori Y Kam; Keith A Garleb; Stephen J Demichele Journal: Clin Gastroenterol Hepatol Date: 2005-04 Impact factor: 11.382
Authors: So Youn Kim; Andrew D Nguyen; Ji-Liang Gao; Philip M Murphy; Brian C Mansfield; Janice Yang Chou Journal: J Biol Chem Date: 2006-08-04 Impact factor: 5.157
Authors: Gepke Visser; Jan Peter Rake; Philippe Labrune; James V Leonard; Shimon Moses; Kurt Ullrich; Udo Wendel; G Peter A Smit Journal: Eur J Pediatr Date: 2002-09-13 Impact factor: 3.183
Authors: D Melis; R Della Casa; R Parini; M Rigoldi; C Cacciapuoti; P Marcolongo; A Benedetti; V Gaudieri; G Andria; G Parenti Journal: Eur J Pediatr Date: 2008-12-09 Impact factor: 3.183
Authors: Katalin M Ross; Iris A Ferrecchia; Kathryn R Dahlberg; Monika Dambska; Patrick T Ryan; David A Weinstein Journal: Adv Nutr Date: 2020-03-01 Impact factor: 8.701
Authors: Dina Khalaf; Heather Bell; David Dale; Vikas Gupta; Hanna Faghfoury; Chantal F Morel; Anne Tierens; David A Weinstein; Jiong Yan; Santhosh Thyagu; Dawn Maze Journal: JIMD Rep Date: 2019-07-23