Diagnosis and Treatment of Myeloproliferative Neoplasms in Germany: First Results from the SAL-MPN Registry

Background Since 2012, the Study Alliance Leukemia (SAL), a group of over 50 clinical trial and research centers (university hospitals, community hospitals, private practice), is establishing a systematic assessment of MPN patients in a German-wide registry, partly supported by Novartis. The main goal of this registry is the evaluation of the epidemiological distribution of clinical characteristics and outcome parameters, to enhance the understanding of MPN disease pathogenesis and disease progression. An important characteristic of our registry is that in addition to large centers, also small institutions are included which allows the collection of real-life data in MPN, due to the fact that these diseases are frequently treated outside large clinical centers in Germany.

Methods The registry is a non-interventional, prospective and multi-centered epidemiological study. Inclusion criteria was the diagnosis of MPN according to WHO criteria (2008). Furthermore a minimum age of 18 and a written informed consent were conditions for entering the registry, independently of therapy regime and comorbidities. In addition to prospective collection of patients, data of patients who died or were lost to follow-up was evaluated once, covered by the central ethics approval. Epidemiological data and clinical characteristics were assessed at date of enrollment and every 12 months for an unlimited period of time. For this analysis only initial data was included. For statistical analysis, descriptive methods were used to describe the data with mean and median including standard deviations, range and/or 95%-confidence intervals.

Results So far, 506 patients have been registered, comprising 243 evaluable patients with a mean age of 55 years (range 21-85). An updated analysis will be presented at the meeting. The proportion of male patients was 52%. The distribution of sex within the entities and other critical parameters are given in Table 1. 62% of all patients were JAK2V617F positive. As expected, complications like thrombosis and thromboembolism were mostly associated with PV and ET (38% and 29%). In 22 % of MPN patients, life-threatening thrombotic or thromboembolic events occurred during the clinical history. Similarly severe bleedings, as defined by a hemoglobin-level decrease > 2 mg/dl, were common in MPN; including 11 % of patients suffering from acute bleeding, most frequently from the upper gastrointestinal tract (56% of all hemorrhages). Acute bleedings occurred most common in PV, followed by PMF patients.

Hydroxyurea was the most frequently administered substance (42%) followed by watch and wait strategies (22%). Other drugs were applied in 36% of all patients.

Conclusions MPN are still associated with life-threatening events, such as thrombosis or severe bleeding. 33% of patients experiencing such an event had already been diagnosed with MPN and thus were already under medical surveillance. Novel strategies to pre-define risk factors and optimal therapeutic strategies are required to improve prevention strategies with the ultimate goal to avoid thromboembolic complications and improve survival and quality of life in MPN.