Work

Too little, too late? The burden of delayed care in NTDT

Year: 2026

Type: editorial

Abstract: In this issue of Blood Advances, Cheng et al 1 report the largest multicenter analysis from the United States specifically examining patients with non-transfusion-dependent thalassemia (NTDT) who were only referred to specialist thalassemia centers during adulthood.Their findings are both eye-opening and concerning.Nearly half of the patients ultimately required initiation of regular transfusion therapy after specialist evaluation, whereas substantial proportions had already developed iron overload, pulmonary hypertension, thrombosis, endocrine complications, or extramedullary hematopoiesis by the time they reached expert care.

Source: Blood Advances

DOI: 10.1182/bloodadvances.2026021248

Authors: Khaled M. Musallam, Thomas D. Coates, Ali T. Taher

Institutions: Khalifa University of Science and Technology, Weill Cornell Medicine, University of Southern California, Children's Hospital Los Angeles, American University of Beirut Medical Center

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,800