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T. Pincez et al.
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The patients who died had received more second-line treatments than the others in the cohort (median 3 vs. 2; P=0.02), including splenectomy, which was more com- mon in this subgroup (56% vs. 20%; P=0.003). Patients who had received more than two second-line treatments had a three-fold increase in the risk of death compared to those who had received two or less (11 of 65 [16.9%] vs. five of 86 [5.8%], P=0.03). At death, 81% of patients were receiving ongoing second-line treatment. The number of second-line treatments (HR 1.3; 95% CI: 1.1–1.6; P=0.004) and severe/recurrent infections (HR 3.4; 95% CI: 1.2–9.7; P=0.02) were independently associated with a higher risk of mortality after 5 years of follow-up.
Discussion
This large follow-up study of pES patients included more than 1,900 patient-years. Over the long term, AIHA and ITP were sustainably controlled in the majority of
patients. Conversely, clinical and biological IM increased in frequency and number with increasing patient age, finally affecting almost all adult patients. The number of cIM was associated with a subsequent increase in the number of second-line treatments received. Mortality was high, frequently occurred while cytopenias were in remis- sion, and most deaths concerned AYA. Two characteristics were associated to mortality: severe or recurrent infec- tions and the number of second-line treatments received. Overall, the age-related clinical picture showed similar trends for all patients, shifting from cytopenia to increased IM, a greater treatment burden, and an increased risk of mortality.
In setting up a nationwide cohort, the CEREVANCE group tried to ensure unbiased patient inclusion in this study. Omitting patients with less than 5 years of follow- up data limited any bias due to short-term follow-up, which probably accounts for many of the discrepancies between previous studies. Indeed, our median follow-up period was more than twice as long as in previous studies
Figure 5. Long-term survival. (A) Survival estimate of patients in terms of time from first cytopenia. At 10-year fol- low-up, survival rates among patients with chronic immune thrombocytopenic purpura (ITP) alone, autoimmune hemolytic anemia (AIHA) alone and pediatric-onset Evans syndrome (pES) were 100%, 99% and 92%, respectively. (B) Mortality is shown in terms of time from first cytopenia, as well as age. Individual values are shown as dots with medians and interquartile ranges shown as lines. (C) Hematological sta- tus at death. CR: complete remission; PR: partial remission; NR: no remission.
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haematologica | 2022; 107(2)