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Pediatric-onset Evans syndrome outcomes
A
B
Figure 4. Second-line treatments. (A) Total number of second-line treatments received according to the age. (B) Number of second-line treatments ongoing according to age.
were herpes zoster (n=17), sinusitis/otitis media (n=15), pneumopathy (n=12), and bronchiectasis (n=11). Patients with infections had more cIM (median, 2 vs. 1; P<0.0001), a higher incidence of hypogammaglobulinemia (53% vs. 28%; P=0.003), and received more second-line treatments (median 3 vs. 1; P<0.0001). Among the 16 patients with severe infection, nine (63%) were receiving an active treat- ment at infection time.
Severe/recurrent infections were independently associ- ated with hypogammaglobulinemia (OR 2.4; 95% CI: 1.10–5.33; P=0.03) and the number of second-line treat- ments (OR 1.34; 95% CI: 1.13–1.71; P=0.002).
Mortality
Sixteen of the 151 patients followed for more than 5 years (10.6%) died, and seven other patients died before the fifth year of follow-up (23 deaths in total, 22 with available data). Patient survival at 5, 10, and 15 years after the first cytopenia was 97%, 92%, and 84%, respectively (Figure 5A). Mortality rates in patients with pES were
higher than those in patients with cITP or AIHA alone (P<0.0001 for both comparisons).
Deaths occurred regularly throughout the follow-up period (median delay after first cytopenia diagnosis, 8.9 years [range, 0.1–24.3 years]) and at a median age of 18.0 years (range, 1.7–31.5 years) (Figure 5B). In the majority of these patients, cytopenia was under control at the time of death: 15 (65%) and 19 (83%) patients had CR or partial remission from ITP and AIHA, respectively (Figure 5C). Mortality was linked to the disease, the treatment, or both in eight (36%), two (9%), and twelve (55%) cases, respec- tively. The most frequent cause of death was infections (n=12 [52%]; Online Supplementary Table S5). Four patients (18%) died of a hemorrhage, and all were less than 13 years of age. The patients who died from a hemorrhage were younger than those who died from an infection (median 10 years vs. 18 years; P=0.03). All of these patients, except for one who died in the first month of a cerebral hemorrhage, had at least one cIM. Eight of the patients (36%) had hypogammaglobulinemia.
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