Patients favor enzyme replacement therapy done at home: Survey
Patients say at-home ERT saves them time and money in traveling to the clinic
Patients prefer receiving enzyme replacement therapy (ERT) at home, citing savings in travel time and cost, over clinical visits, according to a survey of people with Fabry disease and other lysosomal storage disorders in Germany.
“All patients would recommend home-based ERT to other patients” during the two-year follow-up study.
The study, “The patients’ perspective on home-based infusion: A longitudinal observational study in the German healthcare setting for patients with lysosomal storage disorders treated with enzyme replacement therapy,” was published in the journal Molecular Genetics and Metabolism Reports.
ERT for Fabry delivers man-made version of GalA enzyme to bloodstream
Fabry disease is a lysosomal storage disorder — one of more than 70 genetic conditions characterized by malfunction of lysosomes, the so-called “recycling centers” of the cell. In Fabry, mutations in the GLA gene cause a markedly deficient or absent alpha-galactosidase A (GalA) enzyme that results in a toxic buildup of fatty molecules inside cells.
ERT for Fabry delivers a lab-made version of the GalA enzyme directly to the bloodstream. The treatment can be administered at specialized centers or at home by a nurse.
At-home administration makes it easier for patients to carry out their daily routines, which likely helps them to adhere to the treatment. In several countries, including the U.S., ERT is routinely given at home, but in Germany, most patients still receive ERT in specialized centers, hospitals, or doctors’ offices.
Data about how patients feel about these treatment options is limited in Germany. Thus, researchers interviewed a group of 30 patients (mean age 39.9 years; 19 males) with a lysosomal storage disorder who began home-based ERT, after being treated at medical clinics. The study covers the period from January 2019 to June 2021, and included 18 patients with Fabry disease, five with Gaucher disease, six with Pompe disease, and one with mucopolysaccharidosis type 1.
Patients were interviewed before the start of their first home-based ERT and then at regular intervals thereafter — at six, 12, 18, and 24 months. Among those with Fabry with information about their condition, eight had the classic form of the disease with several organs affected, four had predominantly cardiac manifestations, two had a mild form, and one had kidney issues mainly.
Among the overall group of participants, 14 reported other conditions, the most common being hypertension (high blood pressure) and heart disease.
Home-based ERT increases patient satisfaction, and patients perceive it as an equivalent alternative in quality of care, compared to ERT in the center or clinic or at a physician’s office.
Before transitioning to home infusions, 10 patients received their ERT in the hospital, six in a specialized center, and 14 patients as outpatients. On average, 12 reported waiting less than 30 minutes to receive their ERT, and eight patients reported between 30 and 90 minutes of waiting time. One patient reported waiting longer than 90 minutes. The remaining nine patients reported no waiting time.
Infusion took, on average, 2.7 hours (range of 50 minutes to five hours). Most patients (73.3%) arrived at the clinic by car. Travel costs were completely reimbursed by the health insurance for 10% of the patients, with one patient having a co-payment. The majority (86.7%) supported the travel costs themselves or with help from family members.
In the previous three months, six patients had postponed at least one ERT session.
All participants felt they were adequately informed about their condition and treatment options. Twenty-two patients indicated they always felt well cared for by their ERT provider, and four others reported feeling be well cared for most of the time. Two patients reported feeling they were rarely or never well cared for. Overall, 11 patients said an improvement on their care was needed.
Overall, patients rated their treatment satisfaction at baseline at 8.08 points on a scale of 0 to 10 (highest satisfaction).
All patients reported preferring ERT at home, with reasons that included increased comfort, no time lost and no burden associated with traveling, and the ability to coordinate treatment with other appointments. Some patients mentioned how ERT was a burden on their relatives and said the costs of traveling to a specialized center were too high.
With ERT at home, waiting time of more than 30 minutes was reported by a mean of 5% during follow-up, compared to 30% at baseline. However, the proportion of postponed appointments increased slightly.
After starting on home-based treatment, six patients received ERT in a different location than their home. In one case, ERT was administered in a specialized center due to an allergic reaction.
All participants reported preferring home-based ERT
During the study, patients reported feeling adequately informed about home-based ERT and all felt that this improved their ability to cope with the disease. One patient reported no such improvement after 18 months.
Also, all participants reported preferring home-based ERT, with the most common reason being saving time. All but one patient felt safely cared for throughout follow-up.
Fewer patients felt the need to improve their treatment after six months of follow-up when compared to baseline (6.9% vs. 36.7%). None of the participants felt this need at later timepoints. Satisfaction scores increased by a mean of 1.6 points after six months, with a further increase of 0.2 points at 18 months. “Satisfaction seems to remain stable at a high level,” the scientists wrote.
Patients were asked whether home-based ERT is an alternative to center or clinic-based therapy, in terms of quality of care. All patients (except one at six months of follow-up) said home-based ERT was equivalent to hospital administration.
“All patients at each survey time point would recommend home-based ERT to other patients,” the investigators wrote.
Slightly fewer patients needed support from relatives, after they transition to home-based ERT.
Overall, “home-based ERT increases patient satisfaction, and patients perceive it as an equivalent alternative in quality of care, compared to ERT in the center or clinic or at a physician’s office,” the study concluded.
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