Page 185 - Haematologica Vol. 109 - July 2024

ARTICLE - Disease burden of idiopathic MCD
M. Sarmiento Bustamante et al.
dead at the time of data collection (Online Supplementary Table S1). Sixty-one (60%) patients met TAFRO criteria7 and 41 (40%) were classified as having iMCD-NOS. TAFRO patients tended to be more commonly male (TAFRO 62% compared to NOS 49%) and younger (mean [standard de- viation]: 33.0 [17.4] vs. 40.1 [13.9] years) than NOS patients. First, we investigated disease severity at diagnosis and across age ranges. Of the 100 patients with sufficient in- formation to determine disease severity at diagnosis, 77 (77%) initially presented with severe disease. Notably, we found iMCD in individuals of all ages, with the youngest patient in the cohort diagnosed at 1.8 years and the oldest at 74.4 years. We examined severity by age range visually and observed that the majority of patients <30 or >60 years of age presented with severe disease (Figure 1A). When we examined this trend statistically, we found a significant difference among the groups (P=5.3x10-4). Patients <30 and patients >60 years old were more likely to have se- vere disease (94.6% and 90.0%, respectively) as compared to patients between 30 and 60 years old (62.2%) (Online Supplementary Table S2). All but one patient >60 years
old presented with severe disease and all but two of the patients <30 years presented with severe disease, while a large proportion of patients in the 30- to 60-year-old age group presented with mild-moderate disease, which likely affected this result (Figure 1A).
We next investigated the clinical and laboratory abnor- malities present at diagnosis (Online Supplementary Table S3). Specifically, we focused on the degree of anemia and hypoalbuminemia as markers of disease activity at diag- nosis in this cohort. Across all patients, the median (IQR) hemoglobin concentration was 7.8 (6.6-10.5) g/dL and the median (IQR) albumin level was 2.2 (1.8-2.9) g/dL; patients in both TAFRO and NOS subgroups had median levels below the lower limits of normal. Eighty-seven (85.3%) patients had anemia and 81 (79.4%) had hypoalbuminemia at diagnosis (Figure 1B). We also determined the propor- tions of patients overall and by subtype who had clinical abnormalities (Figure 1C, Online Supplementary Table S3). A large majority of patients presented with fluid retention (84%), splenomegaly (72%), and/or hepatomegaly (60%). While TAFRO patients accounted for most patients with
AB
C
Figure 1. Patients with idiopathic multicentric Castleman disease demonstrate severe disease at diagnosis. (A) A large proportion of patients with idiopathic multicentric Castleman disease (iMCD) across all ages had severe disease. Notably, 95% of patients under the age of 30 years presented with severe disease at diagnosis. (B) Within the cohort of 102 iMCD patients, 87 (85.3%) had anemia and 81 (79.4%) had hypoalbuminemia at diagnosis. *Lower limit of normal for hemoglobin in males (12.5 g/dL). †Lower limit of normal for hemoglobin in females (11.5 g/dL). ‡Lower limit of normal for albumin (3.5 g/dL). (C) A large majority of patients presented with clinical symptoms, which ranged from mild to severe. Patients with both clinical subtypes of iMCD (TAFRO and NOS) demonstrated clinical abnormalities. TAFRO: thrombocytopenia, anasarca, fever/elevated C-reactive protein, reticulin fibro- sis/renal failure, and organomegaly; NOS: not otherwise specified.
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