HSCT for MPS disorders (Hunters and Hurlers), (217-2)

10:15 am – 10:45 am Friday, September 16

Hematopoietic Stem Cell Transplant (HSCT) is best known as a treatment modality for hematologic malignancies, some solid tumors, and various autoimmune disorders. It is lesser known as a means to halt progressive symptoms in certain chromosomal inherited disorders such as Hunters and Hurlers disease. Patients with Hunters and Hurlers are born with an inherited x-linked chromosomal disease where sugar molecules are unable to be broken down and digested by the body causing both physical and mental disabilities. There is no cure for these diseases but HSCT is an intensive therapy that has shown promise with these diagnoses, helping to stop progression of symptoms of disease.

Two case studies of children who underwent HSCT at our center, one for Hunter’s and one for Hurler’s, will be presented from both a medical and psychosocial standpoint. How these children first presented, how HSCT affect their disease, the complications they underwent during their HSCT, and the complex psychosocial aspects of receiving chemotherapy as well as an extended hospitalization.

Learning Objectives:

  • The learner will describe the role of Hematopoietic Stem Cell Transplant in the treatment of non-malignant diseases.
  • Identify conditioning regimens and their unique complications for non – malignant diseases.
  • The learner will describe the psychosocial needs of hematopoietic transplant patients and their families when they undergo a complex regiment such as HSCT.