Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression

To join this study, you need to be aged between 3 and 35 years old and have severe aplastic anaemia that hasn't improved with at least one previous treatment. You also need to have a family member who can be a 'haploidentical' donor – someone who is a half-match, like a parent, child, brother, or sister. It's important that your body doesn't have certain antibodies against your donor.

Along with your age and condition, you'll need to be generally well enough for a transplant. This means having good heart and organ function, no serious uncontrolled infections, and no other cancers in the last five years (apart from very minor skin cancers or specific cervical changes). There are also some health insurance and contraception requirements.

However, you won't be able to join if your aplastic anaemia has already developed into certain other serious blood conditions, if you have specific viral infections like HIV or active hepatitis, if you're pregnant or breastfeeding, or if you've recently had certain vaccinations.

If you decide to take part, you will undergo a stem cell transplant from a family donor. This involves preparing your body for the new cells, receiving the donated cells, and then getting a special medicine called cyclophosphamide after the transplant. During the study, you'll have regular hospital visits for check-ups, blood tests, and scans to monitor your health and the success of the transplant. The doctors will closely watch for any side effects or complications. While the intensive part around the transplant might be several weeks, the follow-up period to assess long-term outcomes will continue for some time after that, ensuring your health is monitored closely. You'll discuss the exact schedule with the study team.