Allogeneic hematopoietic stem cell transplantation (HSCT) — from HLA-identical siblings, other HLA-matched donors, or alternative stem cell sources — is pursued in IPEX syndrome with the intent of achieving complete disease resolution and sustained remission. When those goals remain unmet, a further structured management step becomes necessary.

Previous treatment line — goals not achieved

Prior therapy: Allogeneic hematopoietic stem cell transplantation (HSCT).
Unmet goals: Disease resolution; complete IPEX syndrome remission.

For patients who have not achieved remission following HSCT, the next step centres on immunosuppressive therapy — a structured combination approach with a defined primary agent selection. The full regimen, agent choices, and sequencing are detailed in the protocol.

References

DOI: 10.3390/genes12030323

Patients without HSCT are considered for immunosuppressive therapy (IS) and long-term supportive care.
Immunosuppressive therapies are used with a combination of several immunosuppressive agents to control the acute autoimmune phase of IPEX syndrome.
Patients after transplantation also receive immunosuppressive therapy, usually consisting of calcineurin inhibitors, cyclosporine A, sirolimus and tacrolimus, while some patients also receive steroids.
The current study considered rapamycin, a non-calcineurin inhibitor, as the primary choice of immunosuppressive drug, either used alone or in combination.

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IPEX Syndrome: Managing the Acute Autoimmune Phase When Allogeneic Hematopoietic Stem Cell Transplantation Did Not Achieve Remission

References