Startseite Kongressberichte & Archiv 23rd Congress of EHA Thalassemia (2 Presentations) & MDS (1 Presentation)

Thalassemia (2 Presentations) & MDS (1 Presentation)

Author(s): Antonio Piga, et al. ABSTRACT
 
Conclusion
In this phase 2 open-label study, long-term luspatercept treatment in pts with β-thalassemia was generally safe and well tolerated up to 2 years. Clinically relevant measures of luspatercept efficacy were observed in both NTD pts (increased Hgb levels and improved QoL) and TD pts (decreased transfusion burden). 
 
Author(s): Vip Viprakasit, et al. ABSTRACT
 
Conclusion
In the routine clinical care setting, there are critical unmet medical needs for pts with NTDT as they reported lower QoL scores compared with pts with TDT, as captured by two health questionnaires (SF-36v2 and FACT-An), across all domains except one. There is a need for new interventions to treat pts with NTDT and reduce their burden of disease. Significant differences between pt populations from different geographical locations were identified, suggesting social factors had an impact on difference in QoL between pts with TDT and NTDT. Pts from Thailand reported higher QoL scores for domains on the FACT-An questionnaire versus pts from centers in Italy, Greece, and Lebanon; half of the pts with TDT were from the Thai center.
 
PF498 MUTATIONAL AND SUBGROUP ANALYSES OF LOWER-RISK MYELODYSPLASTIC SYNDROMES (MDS) PATIENTS TREATED WITH LUSPATERCEPT: PHASE 2 PACE-MDS STUDY
Author(s): Uwe Platzbecker, et al. ABSTRACT
 
Conclusion
Increased response rates in patients with lower M/E ratios suggest that an expanded erythroid population at baseline may be associated with response to luspatercept, supporting the concept that luspatercept is acting as an erythroid-maturation agent (EMA). Patients with and without the SF3B1 mutation with EPO < 500 IU/L experienced substantial response rates, consistent with earlier results. Additional analyses are ongoing to understand the role of other biomarkers such as mutation status and how they may impact the biology and influence response. As there is not one distinct profile of responder characteristics, this may suggest the possibility that luspatercept may work in a broad range of genotypes and phenotypes.