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Gaucher Disease
Accuracy of chitotriosidase activity and CCL18 concentration in assessing type I Gaucher disease severity. A systematic review with meta-analysis of individual participant data
Ferrata Storti Foundation
Haematologica 2021 Volume 106(2):437-445
Tatiana Raskovalova,1 Patrick B. Deegan,2 Pramod K. Mistry,3 Elena Pavlova,2 Ruby Yang,3 Ari Zimran,4 Juliette Berger,5,6 Céline Bourgne,5,6 Bruno Pereira,7 José Labarère8,9 and Marc G. Berger5,6,10
1Laboratoire d’immunologie, Grenoble University Hospital, Université Grenoble Alpes, Grenoble, France; 2Lysosomal Disorders Unit, Department of Medicine, University of Cambridge, Addenbrooke’s Hospital, Cambridge, UK; 3Pediatric Gastroenterology and Hepatology, Yale University School of Medicine, New Haven, CT, USA; 4Gaucher Clinic, Shaare Zedek Medical Center, Hebrew University-Hadassah Medical School, Jerusalem, Israel; 5CHU Clermont-Ferrand, Hôpital Estaing, Hématologie Biologique, Clermont- Ferrand, France; 6Université Clermont Auvergne, EA 7453 CHELTER, Clermont-Ferrand, France; 7DRCI, CHU Clermont-Ferrand, Clermont-Ferrand, France; 8Quality of Care Unit, INSERM CIC1406, Grenoble University Hospital, Université Grenoble Alpes, Grenoble, France; 9TIMC-IMAG, UMR 5525 CNRS, Université Grenoble Alpes, Grenoble, France and 10CHU Clermont-Ferrand, Service d’Hématologie Clinique Adulte et Thérapie Cellulaire, Hôpital Estaing, Clermont-Ferrand, France
ABSTRACT
Chitotriosidase activity and CCL18 concentration are interchange- ably used for monitoring Gaucher disease (GD) activity, together with clinical assessment. However, comparative studies of these two biomarkers are scarce and of limited sample size. The aim of this systematic review with meta-analysis of individual participant data (IPD) was to compare the accuracy of chitotriosidase activity and CCL18 con- centration for assessing type I GD severity. We identified cross-sectional and prospective cohort studies by searching Medline, EMBASE, and CENTRAL from January 1995 to June 2017, and by contacting research groups. The primary outcome was a composite of liver volume >1.25 multiple of normal (MN), spleen volume >5 MN, hemoglobin concentra- tion <11 g/dL, and platelet count <100x109/L. Overall, IPD included 1,109 observations from 334 patients enrolled in nine primary studies, after excluding 111 patients with undocumented values and 18 patients with deficient chitotriosidase activity. IPD were unavailable for 14 eligible pri- mary studies. The primary outcome was associated with a 5.3-fold (95% Confidence Interval [CI]: 4.2-6.6) and 3.0-fold (95% CI: 2.6-3.6) increase of the geometric mean for chitotriosidase activity and CCL18 concentra- tion, respectively. The corresponding areas under the receiver operating characteristics curves were 0.82 and 0.84 (summary difference, 0.02, 95% CI: -0.02 to 0.05). The addition of chitotriosidase activity did not improve the accuracy of the CCL18 concentration. Estimates remained robust in the sensitivity analysis and consistent across subgroups. Neither the chitotriosidase activity nor the CCL18 concentration varied significantly according to a recent history of bone events among 97 patients. In conclusion, the CCL18 concentration is as accurate as chi- totriosidase activity in assessing hematological and visceral parameters of GD severity and can be measured in all GD patients. This meta-analy- sis supports the use of CCL18 rather than chitotriosidase activity for monito-ring GD activity in routine practice.
Correspondence:
JOSÉ LABARÈRE
jlabarere@chu-grenoble.fr
MARC G. BERGER
mberger@chu-clermontferrand.fr
Received: August 21, 2019. Accepted: January 20, 2020. Pre-published: January 30, 2020.
https://doi.org/10.3324/haematol.2019.236083
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