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T. Toya et al.
ated with age >60 years (P<0.0001), digestive organ (P<0.0001), CNS (P=0.0062), and skeletal involvement (P=0.0072). Cardiovascular (P=0.071), cutaneous (P=0.26), retroperitoneal (P=0.27), respiratory (P=0.28), endocrine involvement (P=0.83) and sex (P=0.89) were not signifi- cantly associated with ECD-related death.
ECD without skeletal lesion
Considering that the prevalence of skeletal lesions in our study was relatively low compared with that of previous studies in Western countries (>95%),5,19 we compared the clinical data of ECD patients with and without skeletal lesions. No obvious clinical difference based on skeletal involvement was observed, except that patients with skeletal lesions had a better outcome than those without (Figure 1C and Table 3).
ECD with digestive involvement
Given that ECD with digestive involvement was associ- ated with worse survival, we examined the characteristics of ECD patients with digestive disease (Table 4). The digestive organs concerned were the liver (50.0%), pan- creas (50.0%), gastrointestinal tract (33.3%), and gallblad- der (16.7%). Causes of death were arrhythmia in one patient with cardiac tamponade, heart and renal failure in
one patient with pericardial effusion and hydronephrosis, sepsis due to rectal perforation in one patient with gas- trointestinal involvement of ECD, and ECD (details unknown) in one patient. An autopsy was carried out in four patients who died during the clinical course of diges- tive disease, and the involvement of ECD cells was histo- logically proven. Histological samples were available in two patients and both were BRAFV600E positive.
No correlation was found between the existence of digestive organ disease and the CRP level at onset (P=0.99), age at onset (P=0.11), sex (P=0.39), year of diag- nosis (P=1), and the presence of specific organ involve- ment.
CNS involvement and associated clinical factors
Considering that the CNS was revealed to be a “risk organ”, based on the result of our analysis and that of a previous study,16 we compared the characteristics of patients with and without CNS disease to determine those were are at risk of CNS involvement (Table 5). Patients with CNS disease displayed significantly higher age at onset than those without (median, 62 [range, 23–76] years vs. 45 [range, 25–70] years; P=0.033). Additionally, the existence of CNS involvement was correlated with the presence of cardiovascular lesions (P=0.015). No statisti-
AB
C
D
Figure 2. Cumulative incidence of ECD-related death. Competing risks models revealed the cumulative incidence of ECD-related death based on (A) age and pres- ence of (B) digestive organs, (C) CNS, (D) skeletal involvement. DO: digestive organ; CNS: central nervous system.
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haematologica | 2018; 103(11)