JOSHA’S CRITICAL REVIEW OF “GENE THERAPY IN PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA” BY A.A. Thompson et al. N Engl J Med 2018; 378:1479- 1493.
Affiliation: Albert-Ludwigs Universität, Freiburg
Keywords: Critical Review, medicine, β-Thalassemia
THESE INTERIM RESULTS OF TWO PHASE I/II TRIALS ARE REMARKABLE. THOMPSON AND COLLEAGUES DEMONSTRATE THE PROGRESS OF CLINICAL GENE THERAPY FOR MONOGENETIC DISEASES WITH A MEDIAN OBSERVATION TIME OF 26 MONTHS. WITH A FURTHER REDUCTION IN TRANSPLANT-ASSISTENSIVE CONDITIONING, THIS POTENTIALLY CURATIVE TREATMENT OF BETA-THALASSEMIA MIGHT BECOME STANDARD OF CARE IN THE FUTURE NOT LIMITED TO HIGH-INCOME COUNTRIES. HOWEVER, LONG-TERM FOLLOW -UP DATE ARE STILL NEEDED. JOSHA’S Conclusion: IMPORTANT PROGRESS – MAY BE APPLIED AS STANDARD OF CARE FOR SELECTED PATIENTS. JOSHA’S Critical Reviews in Oncology and related areas of medicine and science will focus on recently published clinical and non-clinical studies and discoveries. Our editors feel that there is a strong need for expert opinions on studies and discoveries that may potentially impact on patient care. While any judgment will, of course, be a personal and subjective opinion of our editors with all its limitations, we do hope this service will be helpful for our readers and stimulate thoughts and reflections in face of ever more rapid introductions of new drugs and therapeutic strategies in these days. We do encourage comments by our readers!