OBJECTIVES: To synthesize the evidence from longitudinal studies regarding the hematological effects of drugs for Gaucher Disease (GD).

METHODS: Systematic searches were conducted in Medline and Scopus (April 2021). Observational and interventional longitudinal studies, comparative or not, evaluating enzyme replacement therapies (ERTs) and substrate reduction therapies (SRTs) were included. Single mean meta-analyses were conducted using R. The outcomes were change in hemoglobin and platelets values compared to baseline.

RESULTS: The search retrieved 2,246 articles after duplicates removal. Following screening and eligibility assessment, 68 reports were included. Several meta-analyses were conducted for each drug: imiglucerase (6 months to 5 years), velaglucerase alfa (6 months to 7 years), taliglucerase alfa (6 months to 5 years), miglustat (6 months to 4 years), and eliglustat (6 months to 9 years). For the outcome change in hemoglobin values, the only drug that did not result in significantly improvement in naïve patients was miglustat (e.g. 2 years: -0.1 g/dL, 95%CI -0.9; 0.7). In experienced patients (patients who had the disease under control), all drugs maintained the baseline values. For the outcome change in platelet levels, apart from eliglustat, all the other drugs were evaluated in naïve patients and resulted in significantly improvement compared to baseline. In experienced patients, miglustat was the only drug to decrease platelet values (e.g. 2 years: -38.4 10⁹/L, 95%CI -66.1; -10.8). Limitations include the low number of studies in some of the assessed time points and the non-comparative nature of the analyses.

CONCLUSIONS: long-term results showed that ERTs are effective as treatment in both naïve and experienced patients. Miglustat did not significantly improve hemoglobin values in naïve patients and resulted in the decrease of platelet levels in experienced patients. All this evidence should be interpreted considering its limitations and does not replace a well conducted randomized trial.