Why are we doing this research?
Cincinnati Children’s is conducting a research study to evaluate the safety and effectiveness of a treatment regimen combining two previous approaches to treat patients with
HLH.
What will happen in the study?
The study lasts for 8 weeks with the possibility of additional follow up if you or your child proceeds to bone marrow transplant. The following is a list of procedures that will take place during the study:
Before treatment begins, you/your child will have tests done to confirm the diagnosis of HLH and assess the disease. This will includes drawing blood and bone marrow, looking for infections, testing the immune system, and radiology such as an MRI of the brain and a CT scan of the chest and abdomen.
The treatment regimen is divided into two phases
- Induction therapy, in which medication is given to control the overactive immune system
- Continuation therapy, in which medication is given to keep the immune system quiet for those patients waiting to go to bone marrow transplantation
You or your child will be followed closely throughout the 8 weeks on study to test for response to treatment with routine tests, such as measuring the size of the liver and spleen, blood tests, and looking for side effects of the medications.
There are additional blood, bone marrow and spinal fluid that may be taken for research purposes, in addition to the routine tests for HLH treatment. You will have to give additional permission for these to be taken.
Will you/your child be paid to be in this research study?
You will receive no payment or reimbursement for participation in this research study.