GlobalResearch and clinical trials
Stem cell transplantation in Morquio A syndrome: three-year survival of 90.5% in 41 patients at 9 centers
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
An international study pooling the results of stem cell transplantation in Morquio A syndrome has been published. The records of 41 patients who underwent transplantation at nine centers were reviewed retrospectively. The three-year survival rate was 90.5%. Moderate or severe acute GVHD, a reaction of the transplanted cells against the body, developed in 35.5% of patients. Growth in height continued in 6 of the 8 children transplanted before the age of three.
What does it mean for patients and families?
Transplantation may be an alternative to enzyme therapy for some patients, but it carries risks, and long-term results are not yet known. This decision should be made together by the family and the metabolic and transplant teams.
Source
Scientific paper: Allogeneic Hematopoietic Cell Transplantation for Morquio A Syndrome: An International Retrospective Study. Transplantation and Cellular Therapy, 2026. DOI: 10.1016/j.jtct.2026.01.029 · PubMed 41616943
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Morquio A syndrome (MPS IVA)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.