Abstract
BackgroundEven though recent research has achieved significant advancement in the development of therapeutic approaches for Wilson’s diseases (WD), the current treatment options available for WD are still limited, especially for WD patients with neurological symptoms. This study is intended to compare the therapeutic approaches for WD patients with neurological symptoms receiving either combined sodium 2, 3-dimercapto-1-propane sulfonate (DMPS) and zinc treatment or D-penicillamine (DPA) monotherapy as first-line therapy, and identify the more effective therapeutic approach.MethodsThe case records of 158 patients diagnosed with neurological WD were retrospectively analyzed. These patients treated with intravenous DMPS + Zinc and in combination with oral zinc as a maintenance therapy (Group 1) or DPA alone (Group 2) for 1 year. During the period of treatment, the neurological symptoms of the patients were assessed using the Global Assessment Scale (GAS) and Barthel index. The key hematological and biochemical parameters of the patients (such as the levels of aminotransferase, serum ceruloplasmin, 24-h urine copper excretion), as well as adverse effects were recorded and analyzed.ResultsNinety-three patients in Group 1, displayed decreased GAS scores and increased Barthel indexes consistently in comparison with the baseline (P < 0.01). Among them, 82 patients (88.2%) exhibited significant neurological improvement after 1 year, while 8 patients (8.6%) experienced neurological deterioration. Among the 65 patients in Group 2, 37 patients (58.5%) exhibited neurological improvements, while 17 patients (26.2%) experienced neurological deterioration after 1-year follow up. Six patients discontinued their treatment midway due to their exacerbating neurological symptoms. A comprehensive comparison of the effectiveness of the two courses of treatment revealed that patients in group 1 demonstrated a higher improvement ratio (P < 0.01) and lower worsening ratio of the neurological symptoms for the patients (P < 0.01) in comparison to the patients in group 2. Meanwhile, renal function, liver enzyme and blood cell counts remained stabilized in group1.ConclusionsThis study indicates that the combined therapeutic approach of DPMS and zinc may be a preferred first-line therapy in treating the neurological symptoms of WD, in comparison to the treatment with DPA.
Highlights
Even though recent research has achieved significant advancement in the development of therapeutic approaches for Wilson’s diseases (WD), the current treatment options available for WD are still limited, especially for WD patients with neurological symptoms
This study indicates that the combined therapeutic approach of DPMS and zinc may be a preferred first-line therapy in treating the neurological symptoms of WD, in comparison to the treatment with DPA
88.2% of the patients receiving intravenous dimercapto-1propane sulfonate (DMPS) + Zinc as the first-line of therapy showed gradual neurological improvement after 1 year of treatment, while only 58.5% of the patients undergoing the DPA monotherapy group exhibited neurological improvements. This data demonstrates that the combinatorial therapeutic approach using DMPS and zinc as the initial therapy was significantly more effective than DPA monotherapy in case of WD patients manifesting neurological symptoms
Summary
Even though recent research has achieved significant advancement in the development of therapeutic approaches for Wilson’s diseases (WD), the current treatment options available for WD are still limited, especially for WD patients with neurological symptoms. This study is intended to compare the therapeutic approaches for WD patients with neurological symptoms receiving either combined sodium 2, 3-dimercapto-1propane sulfonate (DMPS) and zinc treatment or D-penicillamine (DPA) monotherapy as first-line therapy, and identify the more effective therapeutic approach. Wilson’s disease (WD) is a rare, genetic disorder in which the metabolism of copper is impaired. The accumulation of copper primarily affects the liver, brain, and cornea. This leads to significant liver damage, as well as neurological and psychiatric symptoms. It would be fatal within a few years from the onset of symptoms [2, 3]
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