A retrospective observational single-centre study on the burden of immune thrombocytopenia (ITP).

BACKGROUND: German data on economic consequences of immune thrombocytopenia (ITP) are limited. PATIENTS AND METHODS: A retrospective, observational study based on chart review of adult patients with a confirmed diagnosis of ITP was conducted at a German university hospital. Costs are presented from the hospital perspective. RESULTS: Of 50 eligible patients, 45 could be classified by disease duration: 19 patients < 3 months (38%, newly diagnosed ITP), 12 patients ≥ 3 to < 12 months (24%, persistent ITP), 19 patients ≥ 12 months (38%, chronic ITP). Complications included 85 bleeding events in 43 patients, including 3 intracranial haemorrhages. Documented were 955 outpatient visits in 43 patients (86%) and 92 inpatient hospital admissions in 45 patients (90%). Of the 46 patients (92%) treated, all received corticosteroids, 25 (50%) intravenous immunoglobulin, and 7 (14%) further therapies. 12 patients (24%) underwent splenectomy. Average total direct medical costs (mean (standard deviation)) were 17,091 (18,859) per patient, 12,749 (11,663) in 17 newly diagnosed ITP patients with a 0.88-month (0.65 months) average disease duration, and 29,868 (29,397) in 13 chronic ITP patients with a 33.5-month (16.8 months) average disease duration. Inpatient stays were the main cost drivers. CONCLUSION: These data concerning current healthcare provision for ITP patients in Germany indicate considerable resource consumption and the need for more effective treatment options in individual patients.

Long-term disease history, clinical symptoms, health status, and healthcare utilization in patients suffering from Lambert Eaton myasthenic syndrome: Results of a patient interview survey in Germany.

INTRODUCTION: The burden of disease in Lambert-Eaton myasthenic syndrome (LEMS) patients is unclear. This study focused on the patient's perspective to obtain patient-reported information on clinical symptoms, burden of illness, impact of LEMS on activities of daily living (ADL), and management of LEMS. METHODS: Semi-structured, face-to-face interviews with LEMS patients from two specialized centres in Germany between September and December 2010. RESULTS: Twelve patients participated; mean age 66.7 ± 9.8 years. First symptoms occurred at age 52.5 ± 14.0 years. Mean time between first symptoms and diagnosis was 4.4 ± 6.2 years. Patients reported neuromuscular, cranial, and autonomic symptoms plus general fatigue. Two-thirds of patients reported 10 or more symptoms. The most frequent symptoms were leg weakness (91.7%) and general fatigue (83.3%). Restrictions in ADL were reported always or often in 75% of patients. Over half of the patients (n = 7) reported poor or very poor health status. Mean EQ-5D utility scores were 0.34 ± 0.35, with little day-to-day variation. Patients visited a number of different clinicians; most had been hospitalized at some point in the course of their disease. The most frequent drug treatments were 3,4-diaminopyridine (3,4-DAP) (83.3%) and pyridostigmine (41.5%). The study has several limitations, including small sample size and the potential influence of recall bias. CONCLUSION: LEMS patients report long individual disease histories. Most patients suffer multiple symptoms which are frequently severe and troublesome, and almost all are restricted in ADL with poor health status. There is high utilization of healthcare resources from diagnosis to ongoing treatment. Physicians should be aware of this rare disease to ensure that patients receive an early diagnosis and prompt and appropriate treatment.

Blood component use and associated costs after standard dose chemotherapy--a prospective analysis of routine hospital care in lymphoproliferative disorders and NSCLC in Germany.

PURPOSE: The purpose of this study was to describe blood component (BC) use and respective cost after standard dose chemotherapy (CT) in routine hospital care. METHODS: Analysis of data from a prospective, multicenter, longitudinal, observational study on lymphoproliferative disorder (LPD) and non-small cell lung cancer (NSCLC) patients undergoing first or second line standard dose (immuno-)CT. Data were collected from patient interviews and pre-planned chart reviews. Costs of BC are presented from provider perspective. RESULTS: One hundred eighty patients (n = 85 NSCLC, n = 95 LPD) receiving 189 CT lines/633 CT cycles) were evaluable (mean ± SD age, 59 ± 13.2 years, 68% stage III/IV, 14% Eastern Cooperative Oncology Group ≥ 2). During 11% of cycles, BC were transfused to 27% of patients (n = 49; n = 22 NSCLC, n = 27 LPD). Of 310 transfused units (TU), 68% were red blood cells (RBC). Mean number of TU per cycle with transfusion was 3.3 ± 2.9 (median = 2, range = 2-17) for RBC, 4.8 ± 6.8 (median = 2, range = 1-23) for platelets (PLT) and 12.8 ± 14.6 (median = 8, range = 2-33) for fresh frozen plasma (FFP). Fifteen per cent of RBC units, 60% of PLT units and 92% of FFP in this study were transfused in cycles with sepsis. Mean BC cost per CT line were euro 602 ± 1,458 (median = 135, range = 135-9,385; NSCLC: euro 292 ± 376, median = 135, range = 135-2,124; LPD: euro 1,010 ± 2,137, median = 212, range = 135-9,385, p = 0.2137). For 55% of transfused RBC units, haemoglobin levels on the day of transfusion were 8.0-8.9 g/dl, for 38% <8 g/dl and for 7% ≥ 9 g/dl. Seventy-five per cent of PLT units were transfused at a PLT count <11,000/µl and 21% at 20,000-11,000/µl. CONCLUSIONS: The results reflect the diversity of BC use after standard dose CT. High transfusion need is associated with infectious complications, i.e. sepsis emphasising the need for adequate prophylaxis and further knowledge of baseline risk factors.