It is clear that the survival of patients with inherited and acquired classical hematologic disorders continues to improve. The benefits, however, are not evenly distributed, and some continue to fare better than others. In session 322, Disorders of Coagulation or Fibrinolysis: Clinical and Epidemiological: Practice-changing Information from Datasets and Registries on Bleeding Disorders, Dr. Amanda B. Payne described the trends in mortality in people with hemophilia, based on data reported to the Universal Data Collection (UDC) system, a collaboration between the Centers for Disease Control and Prevention (CDC) and the U.S. Hemophilia Treatment Center Network (USHTCN) showing that improvements in mortality may be limited to male patients and those with severe disease. In session 331, abstract 2496, Dr. Arya Mariam Roy disclosed that, of patients with thrombotic thrombocytopenia purpura (TTP) requiring hospitalization in the United States, Black people, older patients, and those with public insurance had higher mortality rates. This was also the case for those who had sepsis, acute kidney injury, and hypertension. While this study, which is an analysis of the 2017 National Inpatient Sample database, has its limitations, it pulls back the curtain to reveal that health disparities are not being addressed with the same rigor and resources that have historically been given to other prognostic factors.
The advancement of science and medicine should go beyond simply extending survival. To achieve this, the study of long-term effects of disease and associated therapies, with a focus on patient-reported outcomes (PROs), needs to be prioritized. Prevention of major bleeding is a priority in patients with immune thrombocytopenia (ITP). Camelia Vladescu, MSc (session 311, abstract 2447) showed cognitive impairment affects in 50 percent of patients with ITP; hence, bleeding that goes unnoticed, such as occult cerebral microbleeds, is of high clinical relevance. The study presented by Dr. Shruti Chaturvedi (session 331, abstract 138) reported that cognitive impairment also affects about one-third of patients with immune TTP (iTTP), with brain infarction seen on MRI even in those without symptoms of stroke. Further, Dr. Deirdra R. Terrell (session 904 abstract ) presented data showing a high rate of fatigue in iTTP patients. Its degree seems even higher than what has been reported in breast cancer survivors and people who have recovered from severe COVID-19, and similar to those with systemic lupus erythematosus and rheumatoid arthritis. Interventions such as acupuncture for pain management in patients with sickle cell disease, presented by Dr. Ying Wang, encouragingly, although preliminarily appear to have a favorable impact in this population, with corresponding improvement in quality of life.
It is encouraging to see that clinical trials of emerging technologies( including gene therapy) are placing increasing emphasis on PROs. The long-term outcomes of patients with transfusion-dependent β-thalassemia following treatment with betibeglogene autotemcel (autologous gene therapy with a modified β-globin gene), as presented by Dr. Franco Locatelli in session 112, abstract 3665, include improvements in quality of life after two years, with trends toward a positive impact on employment, school attendance, and physical activity.
There is still much to be done. The degree of excitement generated by sharing scientific advances in hematology, should be matched by our commitment to serve people. Only by looking at outcomes through the eyes of those who are experiencing our science can we keep our promise to help people live, not only longer, but better.
Dr. Perez Botero indicated no relevant conflicts of interest.
