- Very many adults with SMA are active in the workforce and living full lives despite their disabilities. Regaining previously lost functionality realistically translates into their everyday life, e.g., they turn part-time jobs into full-time jobs, continue their education or pursue their hobbies," Prof. Anna Kostera-Pruszczyk, head of the Department of Neurology at UCK WUM, said in an interview with Medexpress.
SMA: With pharmacotherapy, we restore patients to their potential
Published June 13, 2023 08:00
For more than four years, Polish patients with SMA have benefited from treatment with nusinersen. How does the therapeutic program monitoring system evaluate the efficacy, safety and effects of this therapy?
Treatment with nusinersen was introduced in Poland under reimbursement in 2019. We are currently treating 820 patients, with a full cross-section of ages and varying degrees of symptom severity at the time of therapy initiation. Through the SMPT system, we continuously track the functional status of our patients on relevant scales. The results are unequivocally positive. Perhaps even above the expectations of those who wondered if they could be achieved especially in adult patients who have been ill for several decades, for whom there were no previous pharmacological treatment options.
Nusinersen has more than 7 years of observations for different age groups in clinical trials and from clinicians' practice. Over these seven years, how does the safety and efficacy profile compare? Do all patients benefit from this therapy?
Spinal muscular atrophy is a highly variable disease. What we know, both from clinical trials of nusinersen and from extended follow-up clinical trials, is very optimistic. Studies show that we get sometimes excellent correction of the phenotype of the clinical picture, depending, of course, on the age of the patient and the condition in which they started treatment. This is true for children who started treatment pre-symptomatic. Today, these are patients from the neonatal screening. In contrast, in the past it was a small group of children. We see not only stabilization, but also improvement in patients who receive chronic therapies, suffering from those forms of the disease in which their condition would deteriorate gradually.
What are the efficacy results and outcomes of treating adults with SMA in Poland with nusinersen therapy based on actual observational data?
Our observations, in terms of adult treatment, have recently been summarized. The 30 months of treatment show a steady, gradual improvement. Of course, she appears in varying degrees, in different patients (not only nusinersen therapy reversed the natural history of the disease), and allowed patients to stabilize their form at the level where they are, and in subsequent years of treatment regain the functions already lost.
How do Polish treatment observations of actual clinical practice in SMA treatment compare to foreign observational studies by Tim Hagenacker and Giorgio Coratti?
The study by Tim Hagenacker's German group and Coratti's meta-analysis referred to groups of patients typically treated with up to 14 months of follow-up. Our observation is therefore much longer. As a result, we can say that we confirm what they saw, which is improvement during the first 14 months of treatment. But we also have evidence that the improvement becomes more pronounced as treatment continues. So our patients have all the time unfulfilled potential that is being returned to them through therapy. I think it's important to keep in mind the fact that very many adults with SMA are active and living full lives despite their disabilities. The recovery of previously lost functionality realistically translates into their everyday life, e.g. they turn part-time work into full-time work, continue their education or pursue their hobbies.
