[Lysosomal Storage Diseases: Challenges in Multiprofessional Patient Care with Enzyme Replacement Therapy]. / Lysosomale Speichererkrankungen: Herausforderungen bei der sektorübergreifenden, multiprofessionellen Patientenversorgung mit Enzymersatztherapie.

Background Due to their rarity studies in (ultra-) rare diseases are difficult. Only for a minority of these diseases causal therapies are available. Development and production of enzyme replacement therapies (ERT) for example are challenging and expensive. The number of patients is low, costs per patient are high. We will focus on the challenges of providing long-term ERT to patients with lysosomal storage diseases (LSD) in an out- and inpatient setting based on a literature search in Pubmed and own experience. Many ERTs for LSDs have a positive cost-benefit ratio. Possible side-effects are severe allergic reactions. ERT is covered by the insurance companies when prescribed by a physician, however they are liable to recourse by the insurance company as the expenses for drugs of the prescribing physician will be above average. In most cases the recourse can be averted if diagnoses of individual patients are disclosed. Intravenous infusion of ERT is not well-regulated in Germany/Austria. Infusion on a ward is safe however often not covered by the insurance companies as patients do not stay overnight. Another option is infusion in a day-care setting, however the lump sum paid for infusion does not cover costs for ERT. On an individual basis, reimbursement for medication (ERT) has to be negotiated with the insurance companies before infusion takes place. Home infusions are feasible, however careful evaluations of the infusion-team and the risk for side-effects have to be performed on an individual basis, legal issues have to be considered. In- and outpatient ERT of patients with LSDs is challenging but feasible after individual evaluation of patient and infusion team.

Ambulatory high-dose methotrexate administration among pediatric osteosarcoma patients in an urban, underserved setting is feasible, safe, and cost-effective.

BACKGROUND: We describe the safety, feasibility, and provide a cost-estimate of outpatient high-dose methotrexate administration (HDMTX) among an urban, underserved population. PROCEDURE: A retrospective analysis of ambulatory HDMTX administration among osteosarcoma patients, at Montefiore Medical Center's Children's Hospital (Bronx, NY) was performed. HDMTX (12 g/m(2)) was given intravenously (IV) over 4 hr after urine alkalinization. Patients were discharged home to continue IV hydration and alkalinization delivered via a home infusion pump. Families were instructed to monitor urine pH overnight and management was adjusted according to our institution's treatment algorithm until MTX level ≤ 0.1 µmol/L. A cost estimate was performed to assess the difference in costs for outpatient versus hypothetical inpatient administrations. RESULTS: Of the 97 ambulatory HDMTX administrations, 99% were successfully completed. One patient failed outpatient administration secondary to home infusion pump malfunction. This patient successfully completed subsequent courses as an outpatient. Most patients (72%) had a MTX level of < 10 µmol/L at 24 hr post-HDMTX. No patients were found to have a MTX level of > 50 µmol/L at 24 hr. About 26% of courses were associated with grade III or IV neutropenia, 4% were associated with grade III or IV thrombocytopenia and 1% were associated with grade III/IV leukopenia. Compared to a hypothetical hospital inpatient stay, the hospital costs for ambulatory HDMTX were an average of $1400 less per cycle. CONCLUSION: Ambulatory HDMTX administration among an underserved, urban population is safe, feasible, and cost-effective.

Out-patient and home-parenteral antibiotic therapy (OHPAT): evaluation of the impact of one year's experience in Tayside.

OBJECTIVE: An out-patient and home parenteral antibiotic therapy programme for the treatment of suitable infections was developed over a four year period. This paper describes the impact of one year's experience of its implementation on various measures of outcome. DESIGN: Each patient treatment has a full integrated care pathway (ICP) and patient satisfaction questionnaire completed. The ICP documents the clinical progress of the patient and incorporates various measures of quality of care on the 101 number of patients treated from April 1998 to March 1999 are presented here. SETTING: Dundee Teaching Hospitals NHS Trust (now Tayside University Hospitals NHS Trust). SUBJECTS: Patients with a range of infections requiring intravenous antibiotics. MAIN MEASURES: Number of patients treated with various infections, clinical and microbiological outcome, drug and vascular access complication rates, impact on drug costs and in-patient bed days, and measurement of patient satisfaction/quality of life. RESULTS: Patients were treated over a 12 month period. 51.5 per cent had skin & soft tissue infections and 22.8 per cent bone & joint sepsis. 57 per cent of patients received out-patient and 34 per cent self or carer administered home therapy. Ninety-four per cent of patients were cured or improved following treatment. Only 7.5 per cent of patients required an unscheduled admission to hospital. Twelve per cent of patients had some type of vascular device related adverse event (partly due to a faulty batch of lines) and six per cent of patients had a drug related reaction. The additional daily cost of drugs was minimal (< 12 Pounds/day) and more than 1,461 bed days have been saved across the Directorates. The patient satisfaction level was high.

Home intravenous therapy in cystic fibrosis: a prospective randomized trial examining clinical, quality of life and cost aspects.

In this study, we set out to determine if home intravenous (i.v.) antibiotic therapy in adult patients with cystic fibrosis (CF) is a feasible, effective and less costly alternative to hospitalization, and to assess the impact of home therapy on quality of life. The study was a prospective, randomized, two-factor mixed design involving adults presenting with respiratory exacerbations of CF. Patients were randomized such that they were discharged home after 2-4 days, or remained in hospital. Seventeen patients had 31 admissions (13 home and 18 hospital). Following 10 days of therapy, there were no significant differences between home or hospital arms with respect to body weight, 12 minute walking distance, sputum weight, pulse oximetry, or improvement in lung function (forced expiratory volume in one second (FEV1), or forced vital capacity (FVC)). Patients who remained in hospital were less fatigued and noted a greater degree of mastery. Patients discharged early noted less disruption to their family life, personal life and sleeping pattern. The total cost for the home therapy arm was approximately half that of the hospital therapy arm. Home intravenous antibiotic therapy in patients with cystic fibrosis was a feasible, cost-effective alternative to receiving therapy in hospital. Although there was no clinical compromise associated with home therapy, there were advantages and disadvantages in terms of quality of life.

Home intravenous antibiotic therapy. A safe and effective alternative to inpatient care.

OBJECTIVE: To assess the practicality, safety, cost effectiveness and outcome of receiving intravenous antibiotics at home. METHODS: Patients with serious bacterial infections requiring parenteral antibiotic therapy were enrolled in a pilot program to receive treatment at home. Antibiotics were premixed in the hospital pharmacy and administered by the Royal District Nursing Service, and medical back-up was provided. RESULTS: Twenty patients (mean age, 58 years; range, 19-84 years) received 21 courses of intravenous antibiotics at home (mean duration +/- SD, 26 +/- 9 days; range, 11-44 days). Conditions treated included osteomyelitis (10 patients), endocarditis (5), vascular graft and pacemaker sepsis (4), and chronic cellulitis (1). Treatment at home was well tolerated with no significant complications, and cure was achieved in 18 of the 20 patients. It was both efficient and cost effective, with a mean benefit in treatment costs between home and the equivalent inpatient therapy of at least $112 per day for the 538 days that home therapy was provided. Moreover, the reduced bed use could allow an additional hospital throughput of between 86 and 107 patients annually. CONCLUSIONS: Home intravenous antibiotic therapy is safe, effective and well tolerated. It allows more efficient inpatient care and reduces total treatment costs in an important subpopulation of patients.