This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.
Laronidase Enzyme Replacement Therapy will be performed using laronidase once a week for 12 weeks prior to hematopoietic stem cell transplantation and for 8 weeks post-transplant to reduce pulmonary complications.
Study Type
OBSERVATIONAL
Enrollment
20
Administered 0.58 mg/kg/dose intravenously (IV) once a week beginning 12 weeks before planned hematopoietic stem cell transplant (HSCT) and resume same dosing regimen for 8 weeks after HSCT.
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
Overall Survival
Patients alive at 1 year post transplantation.
Time frame: At 1 Year
Incidence of Engraftment
The incidence of donor engraftment will be estimated by taking the simple proportion of patients achieving donor engraftment over the number of evaluable patients. Donor engraftment will be defined as achieving an absolute neutrophil count ≥ 5x10\^8/kg for three consecutive days before day 42 and maintenance of \>10% donor chimerism through one year post transplant or death.
Time frame: 1 Year Post Transplant
Incidence of Grade III-IV Acute Graft Versus Host Disease
Cumulative incidence will be used to estimate grade III-IV acute GvHD, treating death as a competing risk.
Time frame: Day 100
Proportion of patients in need of ventilator support
Count of patients using ventilator by 1 year.
Time frame: 1 Year
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