Prognostic Impact of Organomegaly in Mastocytosis: An Analysis of the European Competence Network on Mastocytosis

• Published in: The journal of allergy and clinical immunology in practice (Cambridge, MA) Vol. 11; no. 2; pp. 581 - 590.e5
• Main Authors: Lübke, Johannes, Schwaab, Juliana, Christen, Deborah, Elberink, Hanneke Oude, Span, Bart, Niedoszytko, Marek, Gorska, Aleksandra, Lange, Magdalena, Gleixner, Karoline V., Hadzijusufovic, Emir, Solomianyi, Oleksii, Angelova-Fischer, Irena, Zanotti, Roberta, Bonifacio, Massimiliano, Bonadonna, Patrizia, Shoumariyeh, Khalid, von Bubnoff, Nikolas, Müller, Sabine, Perkins, Cecelia, Elena, Chiara, Malcovati, Luca, Hagglund, Hans, Mattsson, Mattias, Parente, Roberta, Varkonyi, Judit, Fortina, Anna Belloni, Caroppo, Francesca, Zink, Alexander, Brockow, Knut, Breynaert, Christine, Bullens, Dominique, Yavuz, Akif Selim, Doubek, Michael, Sabato, Vito, Schug, Tanja, Niederwieser, Dietger, Hartmann, Karin, Triggiani, Massimo, Gotlib, Jason, Hermine, Olivier, Arock, Michel, Kluin-Nelemans, Hanneke C., Panse, Jens, Sperr, Wolfgang R., Valent, Peter, Reiter, Andreas, Jawhar, Mohamad
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01-02-2023
• Subjects: Disease Progression; Hepatomegaly; Humans; Lymphadenopathy; Mastocytosis; Mastocytosis - diagnosis; Mastocytosis, Cutaneous; Mastocytosis, Systemic - diagnosis; Mastocytosis, Systemic - epidemiology; Medicin och hälsovetenskap; Organomegaly; Prognosis; Splenomegaly; Systemic mastocytosis; SM-AHN; Mastocytosis; Splenomegaly; ISM; OS; Organomegaly; MRI; AHN; CM; HR; BM; SSM; AdvSM; Systemic mastocytosis; Lymphadenopathy; Hepatomegaly; MC; MIS; MCL; SM; ASM; PFS; ECNM
• ISSN: 2213-2198; 2213-2201; 2213-2201
• DOI: 10.1016/j.jaip.2022.10.051

Organomegaly, including splenomegaly, hepatomegaly, and/or lymphadenopathy, are important diagnostic and prognostic features in patients with cutaneous mastocytosis (CM) or systemic mastocytosis (SM).
To investigate the prevalence and prognostic impact of 1 or more organomegalies on clinical course and survival in patients with CM/SM.
Therefore, 3155 patients with CM (n = 1002 [32%]) or SM (n = 2153 [68%]) enrolled within the registry of the European Competence Network on Mastocytosis were analyzed.
Overall survival (OS) was adversely affected by the number of organomegalies (OS: #0 vs #1 hazard ratio [HR], 4.9; 95% CI, 3.4-7.1, P < .001; #1 vs #2 HR, 2.1, 95% CI, 1.4-3.1, P < .001; #2 vs #3 HR, 1.7, 95% CI, 1.2-2.5, P = .004). Lymphadenopathy was frequently detected in patients with smoldering SM (SSM, 18 of 60 [30%]) or advanced SM (AdvSM, 137 of 344 [40%]). Its presence confered an inferior outcome in patients with AdvSM compared with patients with AdvSM without lymphadenopathy (median OS, 3.8 vs 2.6 years; HR, 1.6; 95% CI, 1.2-2.2; P = .003). OS was not different between patients having organomegaly with either ISM or SSM (median, 25.5 years vs not reached; P = .435). At time of disease progression, a new occurrence of any organomegaly was observed in 17 of 40 (43%) patients with ISM, 4 of 10 (40%) patients with SSM, and 33 of 86 (38%) patients with AdvSM, respectively.
Organomegalies including lymphadenopathy are often found in SSM and AdvSM. ISM with organomegaly has a similar course and prognosis compared with SSM. The number of organomegalies is adversely associated with OS. A new occurrence of organomegaly in all variants of SM may indicate disease progression.