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Scientific Oral Presentation

Pediatric Otolaryngology

Auditory Outcomes After Hematologic Treatment in Children with Hurler Syndrome: A 10-year Retrospective Review

Tuesday, October 20, 2026
8:45 AM - 8:51 AM PT
Location: 409AB

    Presenting Author(s)

  • TC

    Thamiris D.D Cabral, MD

    Research Fellow
    Department of Otolaryngology, University of Minnesota, MN, USA
    Rio De Janeiro, Rio de Janeiro, Brazil

    • Co-Author(s)

  • Lauren Buchholz, BA

    Medical Student
    University of Minnesota, Children's Minnesota Hospital
    Minneapolis, Minnesota, United States

  • Evan N. Herschi, BA

    Medical Student
    University of Minnesota Medical School, Masonic Children's Hospital
    Prior Lake, MN, United States

  • PO

    Paul Orchard, MD

    MD
    Department of Hematology, University of Minnesota, MN, USA, United States

    • Senior Author(s)

  • LJ

    Luke A. Jakubowski, MD

    Dr
    Department of Otolaryngology, University of Minnesota, Children's Minnesota Hospital, MN, USA
    Minneapolis, MN, United States

Disclosure(s):
Thamiris D.D. D.D Cabral, MD:

faculty for this accredited education activity has no relationship(s) with ineligible companies to disclose.

Evan N. Herschi, BA:

faculty for this accredited education activity has no relationship(s) with ineligible companies to disclose.

Introduction: Although hematopoietic stem cell transplantation (HSCT) improves systemic manifestations in Hurler Syndrome (HS) patients, its effect on hearing remains poorly defined. Data on the influence of age at therapy initiation, enzyme replacement therapy (ERT) duration, and newborn hearing screening (NHS) have not been reported previously.

Methods: To evaluate changes in best-ear and worst-ear pure-tone average (PTA) and speech reception threshold (SRT), and otoacoustic emissions (OAEs) before and after therapy, HSCT or gene therapy. A retrospective chart review of HS pediatric patients. Outcomes included change in OAE and best-ear and worst-ear assessment of PTA and SRT. Fisher’s exact and Wilcoxon tests were used. Regression analyses were performed.

Results: Sixty-four patients were included (39 females), with mean age at therapy initiation of 18.07 ± 34.71 months. HL occurred in 25% of NHS-negative and 55.6% of NHS-positive patients with no significant association (p=0.205). Early post-transplant OAE showed significant improvement (p=0.019), which was not sustained long-term (p=0.122). Neither ERT treatment (p=0.397) nor age at therapy initiation (p=0.837) was significantly associated with early OAE change. Best-ear hearing improved significantly for PTA (mean -14.64 +- 9.49, p= 0.0009) and SRT (mean -9.58 +- 12.87, p= 0.025), whereas worst-ear improved modestly in PTA (mean -2.95 +- 11.50, p=0.1309) and SRT (mean -3.33 +- 14.97, p=0.2637).

Conclusions: Hematologic therapy was not associated with long-term deterioration of best-ear hearing and showed a favorable profile for the worst ear. Although early OAE improvement was not sustained, larger prospective studies with longitudinal assessments are needed to clarify long-term auditory outcomes and predictors of response.