Eculizumab is generally well tolerated, although the following side effects have been reported: stomach upsets, cough, loss of appetite, runny nose, sore throat, headache, dizziness, tiredness, altered taste, pain in the joints or muscles, relatively few platelets in blood (thrombocytopenia), low white blood cell count (leukopenia), hair loss, itching, rash and flu-like symptoms. Allergic reactions are uncommon. People with STEC HUS may develop many of these symptoms during the usual course of their illness.
Special warning – Please note
Importantly, eculizumab treatment may reduce your child’s natural resistance to infections, especially against “meningococcus”, an organism that causes meningitis (infection of the linings of the brain) and septicaemia (blood infection). The risk of this infection is low – from information about patients treated with eculizumab for other conditions we estimate the risk of developing meningococcal infection is around one in 4,000 per patient receiving eculizumab in the trial.
To ensure the risk of meningococcal infection is as low as possible, your child will be vaccinated against meningococcus. Since some younger children will have received a particular meningococcal vaccination as part of the UK vaccination schedule (“meningitis B vaccine”), the research team will liaise with your child’s GP to determine which vaccines are necessary. Your child will also receive 8 weeks of penicillin (given twice daily by mouth, or an alternative if they are allergic to penicillin) and the first dose will be given before receiving the trial treatment (eculizumab or placebo). To make this trial the best possible test of which treatment is better, it is important to keep all treatments the same in both groups except eculizumab. Therefore people in both groups will receive vaccination and penicillin/alternative.
Side effects of meningococcal vaccination include sleepiness, headache, nausea, vomiting, diarrhoea, rash, pains in muscles and joints, irritability, injection site pain and redness. Side effects of penicillin include stomach upsets, stomach ache and allergy (rash, cough, wheeze, tightness in throat).
We will also give you information to recognise the signs and symptoms of meningococcal infection and advice about what to do in the unlikely event that you suspect your child may have meningococcal infection.
Potential harm to the unborn child
For Girls
The effect of eculizumab on the development of an embryo, foetus or unborn child is currently unknown. Therefore, if your child is a sexually active female of child-bearing potential, she must agree to not become pregnant for 6 months from enrolment into the trial. In addition, your daughter must agree to use an effective and reliable method of birth control that has been agreed upon by the trial doctor for 6 months from enrolment into the trial. Some birth control methods that are acceptable for this trial are abstinence, the consistent use of an approved oral contraceptive (birth control pill or “the pill”), an intrauterine device (IUD), hormonal implants, contraceptive injection or a double barrier method (diaphragm with spermicidal gel or condom with contraceptive foam). The trial doctor will need to approve the method chosen. If your daughter should become pregnant during the trial, the trial doctor should immediately be notified. No further doses of trial drug will be given and the progress of her pregnancy will be monitored until the outcome of the pregnancy is known.
For Boys
If your son is a sexually active male, he must inform his partner(s) (if she/they are female of child-bearing potential), that the effects of eculizumab on sperm are unknown. Your son or his partner(s) should use acceptable methods of birth control (as above), to avoid his partner(s) becoming pregnant for 6 months from enrolment into the trial.