Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

Marsha Treadwell; Paul R. Harmatz; Barbara K. Burton; John J. Mitchell; Nicole Muschol; Simon A. Jones; Gregory M. Pastores; Heather A. Lau; Rebecca Sparkes; V. Reid Sutton; Bianca Meesen; Christine A. Haller; Adam J. Shaywitz; Jeffrey I. Gold

doi:10.1177/2326409817718850

Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

Marsha Treadwell^*, Paul R. Harmatz, Barbara K. Burton, John J. Mitchell, Nicole Muschol, Simon A. Jones, Gregory M. Pastores, Heather A. Lau, Rebecca Sparkes, V. Reid Sutton, Bianca Meesen, Christine A. Haller, Adam J. Shaywitz, Jeffrey I. Gold

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › peer-review

5 Scopus citations

Abstract

Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-reported pain, of 52 weeks of treatment with elosulfase alfa (at a dose of 2.0 or 4.0 mg/kg/week) in patients with Morquio A syndrome (≥7 years old). Assessment of pain at baseline revealed that patients (N = 25) had a mean number of pain locations of 5.7, mean pain intensity score of 4.6 (indicative of medium pain), and a mean number of selected pain descriptors of 7.4 words. Treatment with elosulfase alfa improved subjective pain score (reduced to 3.2), pain locations (reduced by a mean of 1 location), and pain descriptor words (reduced to 4.9 words) over 1 year (52 weeks), suggesting that elosulfase alfa can reduce pain in some patients with Morquio A.

Original language	English (US)
Journal	Journal of Inborn Errors of Metabolism and Screening
Volume	5
DOIs	https://doi.org/10.1177/2326409817718850
State	Published - Jul 21 2017

Keywords

chronic pain
elosulfase alfa
enzyme replacement therapy
galactosamine-6-sulfatase
lysosomal storage diseases
Morquio A syndrome
mucopolysaccharidosis IVA

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Endocrinology, Diabetes and Metabolism
Genetics(clinical)

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1177/2326409817718850

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Treadwell, M., Harmatz, P. R., Burton, B. K., Mitchell, J. J., Muschol, N., Jones, S. A., Pastores, G. M., Lau, H. A., Sparkes, R., Sutton, V. R., Meesen, B., Haller, C. A., Shaywitz, A. J., & Gold, J. I. (2017). Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study. Journal of Inborn Errors of Metabolism and Screening, 5. https://doi.org/10.1177/2326409817718850

@article{99969aad33cf4a1f87a7c51f73d1c7b0,

title = "Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study",

abstract = "Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-reported pain, of 52 weeks of treatment with elosulfase alfa (at a dose of 2.0 or 4.0 mg/kg/week) in patients with Morquio A syndrome (≥7 years old). Assessment of pain at baseline revealed that patients (N = 25) had a mean number of pain locations of 5.7, mean pain intensity score of 4.6 (indicative of medium pain), and a mean number of selected pain descriptors of 7.4 words. Treatment with elosulfase alfa improved subjective pain score (reduced to 3.2), pain locations (reduced by a mean of 1 location), and pain descriptor words (reduced to 4.9 words) over 1 year (52 weeks), suggesting that elosulfase alfa can reduce pain in some patients with Morquio A.",

keywords = "chronic pain, elosulfase alfa, enzyme replacement therapy, galactosamine-6-sulfatase, lysosomal storage diseases, Morquio A syndrome, mucopolysaccharidosis IVA",

author = "Marsha Treadwell and Harmatz, {Paul R.} and Burton, {Barbara K.} and Mitchell, {John J.} and Nicole Muschol and Jones, {Simon A.} and Pastores, {Gregory M.} and Lau, {Heather A.} and Rebecca Sparkes and Sutton, {V. Reid} and Bianca Meesen and Haller, {Christine A.} and Shaywitz, {Adam J.} and Gold, {Jeffrey I.}",

note = "Publisher Copyright: {\textcopyright} The Author(s) 2017.",

year = "2017",

month = jul,

day = "21",

doi = "10.1177/2326409817718850",

language = "English (US)",

volume = "5",

journal = "Journal of Inborn Errors of Metabolism and Screening",

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Treadwell, M, Harmatz, PR, Burton, BK, Mitchell, JJ, Muschol, N, Jones, SA, Pastores, GM, Lau, HA, Sparkes, R, Sutton, VR, Meesen, B, Haller, CA, Shaywitz, AJ & Gold, JI 2017, 'Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study', Journal of Inborn Errors of Metabolism and Screening, vol. 5. https://doi.org/10.1177/2326409817718850

TY - JOUR

T1 - Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks

T2 - MOR-008: A Randomized, Double-Blind, Pilot Study

AU - Treadwell, Marsha

AU - Harmatz, Paul R.

AU - Burton, Barbara K.

AU - Mitchell, John J.

AU - Muschol, Nicole

AU - Jones, Simon A.

AU - Pastores, Gregory M.

AU - Lau, Heather A.

AU - Sparkes, Rebecca

AU - Sutton, V. Reid

AU - Meesen, Bianca

AU - Haller, Christine A.

AU - Shaywitz, Adam J.

AU - Gold, Jeffrey I.

PY - 2017/7/21

Y1 - 2017/7/21

N2 - Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-reported pain, of 52 weeks of treatment with elosulfase alfa (at a dose of 2.0 or 4.0 mg/kg/week) in patients with Morquio A syndrome (≥7 years old). Assessment of pain at baseline revealed that patients (N = 25) had a mean number of pain locations of 5.7, mean pain intensity score of 4.6 (indicative of medium pain), and a mean number of selected pain descriptors of 7.4 words. Treatment with elosulfase alfa improved subjective pain score (reduced to 3.2), pain locations (reduced by a mean of 1 location), and pain descriptor words (reduced to 4.9 words) over 1 year (52 weeks), suggesting that elosulfase alfa can reduce pain in some patients with Morquio A.

AB - Patients with mucopolysaccharidosis (MPS), and Morquio A syndrome (MPS IVA) in particular, often report substantial pain burden. MOR-008 was a randomized, double-blind, pilot study assessing the safety and efficacy, including impact on patient-reported pain, of 52 weeks of treatment with elosulfase alfa (at a dose of 2.0 or 4.0 mg/kg/week) in patients with Morquio A syndrome (≥7 years old). Assessment of pain at baseline revealed that patients (N = 25) had a mean number of pain locations of 5.7, mean pain intensity score of 4.6 (indicative of medium pain), and a mean number of selected pain descriptors of 7.4 words. Treatment with elosulfase alfa improved subjective pain score (reduced to 3.2), pain locations (reduced by a mean of 1 location), and pain descriptor words (reduced to 4.9 words) over 1 year (52 weeks), suggesting that elosulfase alfa can reduce pain in some patients with Morquio A.

KW - chronic pain

KW - elosulfase alfa

KW - enzyme replacement therapy

KW - galactosamine-6-sulfatase

KW - lysosomal storage diseases

KW - Morquio A syndrome

KW - mucopolysaccharidosis IVA

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UR - http://www.scopus.com/inward/citedby.url?scp=85067309363&partnerID=8YFLogxK

U2 - 10.1177/2326409817718850

DO - 10.1177/2326409817718850

M3 - Article

AN - SCOPUS:85067309363

SN - 2326-4098

VL - 5

JO - Journal of Inborn Errors of Metabolism and Screening

JF - Journal of Inborn Errors of Metabolism and Screening

ER -

Impact of Elosulfase Alfa on Pain in Patients with Morquio A Syndrome over 52 Weeks: MOR-008: A Randomized, Double-Blind, Pilot Study

Abstract

Keywords

ASJC Scopus subject areas

UN SDGs

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