[Clinical long-term results of H.E.L.P.-apheresis]

The heparin-induced extracorporeal LDL-precipitation (H.E.L.P.) procedure was approved by the German Health Authorities with the requirement of performing a five year postmarketing surveillance (PMS) study to document safety and efficiency data. A postmarketing surveillance resembles a patient registry including a patient population inhomogeneous to a certain degree with respect to clinical diagnosis and stage of the disease, respectively. Compared to clinical studies a patient registry differs by the length of regular documentation due to the fact that new patients can be recruited at any time during the 5 years of registry documentation. These restrictions must be considered to correctly interpret the PMS data presented. During the study period, a total of 628 patients were recruited at 82 HELP-treatment facilities. The mean pre-treatment LDL value was 276 mg/dl and the mean HDL value 45 mg/dl corresponding to an atherogenic index of 6.1. In addition to the regular HELP treatment, most of the patients were treated by diet and statins (88.5%) or other lipid-lowering drugs. Although 90% of all patients had been treated by maximal conventional lipid-lowering drug treatment, 42.2% of all documented patients had LDL plasma values >250 mg/dl; 19% of all documented patients exerted LDL values of >400 mg/dl and were therefore classified as homozygous FH patients. The mean surveillance time was 2 years. For 243 patients documentation was stopped early due to varying reasons: 19 patients passed away during the postmarketing surveillance; in 7 cases HELP treatment was discontinued because of adverse effects, and 78 patients changed the LDL apheresis treatment device and were therefore lost for the PMS. Lacking patient compliance and refusal of further reimbursement of the treatment costs were reasons in 28 patients, and medical reasons were documented for another 111 patients for early termination of apheresis treatment. From 58 991 documented single HELP-treatment procedures, the treated plasma volume was available as a measure for treatment efficiency. The mean treated plasma volume was 2.8 l; in 83.9% of all documented treatments a minimum of >2.5 l was treated. The mean treatment interval was 9.9 days. Out of 628 patients, 158 complained of angina before regular HELP treatment. At the final examination, the majority of these patients (87%) reported marked improvement, similar to self-reported improvement of dyspnea on exertion. The incidence of cardiac events including death, myocardial infarction, angioplasty and CABG was lower in patients with LDL >250 mg/dl (12.3%) as compared to patients with LDL <250 mg/dl (17%). For all patients, the relative rate of events was 16%. One further aim of the PMS study was the documentation of adverse effects of regular HELP treatment using a questionnaire. During five years of PMS, 2734 (4.6%) adverse effects during 59 121 documented single HELP treatments in 622 patients were recorded. To define adverse effects directly related to the HELP procedure, adverse effects of the concomitant drugs administered require careful consideration. Therefore, the incidence of adverse effects directly attributable to HELP does not exceed 1-2%.