Wonderful medicine for rare diseases! Is Eculizumab effective?

For paroxysmal nocturnal hemoglobinuria (PNH), eculizumab was compared with placebo in a major study involving 87 adults with PNH who had received at least four blood transfusions for anemia in the past year. The main measures of effectiveness were eculizumab's effect on hemoglobin blood levels and the need for transfusions. Hemoglobin is the protein in red blood cells that carries oxygen throughout the body. In people with PNH, the breakdown of red blood cells causes lower hemoglobin levels. After 26 weeks of eculizumab treatment, 49% of patients had stable hemoglobin levels and did not require red blood cell transfusions.

In a study of7 children with PNH who had received at least one blood transfusion in the past two years, all received eculizumab. Six out of seven patients did not require any red blood cell transfusions and showed improvement in hemoglobin levels during 12 weeks of eculizumab treatment. A registry study of transfusion-naïve PNH patients looked at blood levels of lactate dehydrogenase (LDH). As the breakdown of red blood cells increases, LDH levels rise. The study found that treatment with eculizumab for 6 months resulted in a clinically meaningful reduction in LDH levels, indicating reduced breakdown of red blood cells.

For atypical hemolytic uremic syndrome (aHUS), eculizumab was studied in three major studies involving 67 patients. The first study involved 17 patients with aHUS who were unresponsive or untreatable to plasma exchange or transfusion. Treatment with eculizumab resulted in an increase in platelet counts in 82% of patients, with platelet counts rising to normal levels in patients who started with low platelet counts (87%). Additionally, 76% of patients achieved hematological normalization (platelet and lactate dehydrogenase levels within normal limits).

The second study involved20 aHUS patients who had already received plasma exchange or infusion. As a result, 80% of the patients no longer required plasma exchange, infusion or dialysis, and 90% of the patients achieved hematological normalization after eculizumab treatment. The third study involved 30 patients with aHUS who had received at least one dose of eculizumab. Treatment increased platelet counts to normal levels in 83% of patients, while 77% had initially low platelet counts.

Eculizumab was compared with placebo in a major study involving126 adults with generalized myasthenia gravis (gMG) who had previously received failed standard treatments. According to a standard scoring system, eculizumab treatment improved the patient's symptoms and ability to perform daily activities. After 26 weeks, eculizumab scores decreased by 4.7 points. A 2-point decrease in score indicates a clinically significant improvement in the patient's condition. Similar results were found in children. A major study of 11 children over 12 years old showed that eculizumab improved symptoms and patients' ability to perform daily activities by 5.2 and 5.8 points after 12 and 26 weeks of treatment, respectively. Based on these results, the drug is expected to work in a similar way in children ages 6 to 12.

For neuromyelitis optica (NMOSD), eculizumab was compared with placebo in a major study involving 143 adults with NMOSD who had relapsed disease. The primary measure of effectiveness is the time it takes for a certain number of patients to experience relapse. Three percent of patients who received eculizumab relapsed after an average of about 22 months, compared with 43% of patients who received placebo after an average of about nine months.