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T. Pincez et al.
with age. At 10 compared to 20 years old, 37% and 74% of patients had at least one cIM and 9% and 34% of patients had at least two cIM, respectively (P<0.001 for both comparisons; Figure 2B).
The most common cIM categories were lymphoprolifer- ation (n=71), dermatological (n= 26), gastrointestinal/hepat- ic (n=23) and pneumological manifestations (n=16, Figure 3; Online Supplementary Figure S2B). The most frequent cIM diagnosis are shown in Table 2. Thirteen patients devel- oped a neurological manifestation as previously described.10 Four patients had a hematological malignancy (age at diag- nosis): Hodgkin lymphoma (16 years), juvenile myelomonocytic leukemia (20 years), large granular lym- phocytic leukemia (21 years) and angioimmunoblastic T- cell lymphoma (29 years). Older age at ES diagnosis (HR 1.09; 95% CI: 1.01–1.17; P=0.02), cIM/cancer in a first- degree relative (HR 1.64; 95% CI: 1.1–2.4; P=0.006), and the presence of AIN were independently associated with the number of cIM (HR 2.41; 95% CI: 1.5–3.8; P=0.0002).
Biological IM (bIM) were diagnosed in 101 of 151 patients (67%), and the frequency of bIM also increased with the age (Figure 2C). Hypogammaglobulinemia was
the most frequently diagnosed bIM (n=54), including 44 cases diagnosed prior to any anti-CD20 treatment. Among those 54 patients, 25 (46%) received immunoglobulin replacement therapy. SLE and ALPS biomarkers were present (regardless of whether patients met the diagnostic criteria) in 42 and 24 patients, respectively. At 10 and 20 years of age, 39% and 75% of patients had at least one bIM, respectively (P<0.001).
Patients with bIM were more likely to have cIM (79% vs. 40%; P<0.001), and patients with cIM were more likely to have bIM (80% vs. 41%; P<0.001) but the correlation between the number of bIM and cIM was low (r=0.27; P<0.001).
Secondary pediatric-onset Evans syndrome
In 37 patients (24.5%), pES eventually revealed a SLE or a PID unknown at cytopenia onset.
Eleven patients (7.3%) eventually met the SLE SLICC diagnostic criteria.16 These patients were older at first cytopenia (median age 13 years vs. 5 years; P=0.007) and almost exclusively female (one of 88 males [1%] and ten of 63 females [16%]); P<0.001).
A
Figure 1. Hematological outcomes.
(A) Cumulative incidence of patients achieving a sustained complete remission (CR). Among the 23 patients without sustained CR for autoimmune hemolytic anemia (AIHA), four (17%) had achieved sus- tained CR for immune thrombocy- topenic purpura (ITP). Conversely, among the 32 patients without sus- tained CR for ITP, 13 (40%) had achieved sustained CR for AIHA. (B) Percentage of patients with a sus- tained complete remission according to age.
B
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haematologica | 2022; 107(2)