For Medical Professionals

[GinJones]

Looking for the latest in hypophosphatemia research and news? We've got the links here, or you can sign up to receive the updates in a newsletter (once or twice a year) with the latest news and research citations: http://eepurl.com/bVz6O5

Recent news:

Phase 3 clinical trials (adults with XLH) have begun for KRN23, a recombinant human immunoglobulin G isotype 1 (IgG1) monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23). For more information (two separate trials): https://clinicaltrials.gov/ct2/show/NCT02526160?term=XLH&rank=1 or https://clinicaltrials.gov/ct2/show/NCT02537431?term=XLH&rank=2

A Phase 2 clinical trial has begun for adults with TIO, also for KRN23, a recombinant human immunoglobulin G isotype 1 (IgG1) monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23). https://clinicaltrials.gov/ct2/show/NCT02304367?term=TIO&rank=1

Phase 3 pediatric trials for the same treatment are expected later in 2016.


For basic information on hypophosphatemia, check out:

A CLINICIAN'S GUIDE TO X-LINKED HYPOPHOSPHATEMIA, by Thomas O. Carpenter, Erik A. Imel, Ingrid A. Holm, Suzanne M. Jan de Beur, and Karl L. Insogna (2011), available for download here: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3157040/

THERAPEUTIC MANAGEMENT OF HYPOPHOSPHATEMIC RICKETS FROM INFANCY TO ADULTHOOD. Linglart A1, Biosse-Duplan M, Briot K, Chaussain C, Esterle L, Guillaume-Czitrom S, Kamenicky P, Nevoux J, Prié D, Rothenbuhler A, Wicart P, Harvengt P., Endocr Connect. 2014 Mar 14;3(1):R13-30. doi: 10.1530/EC-13-0103. Print 2014. PMID: 24550322   http://www.endocrineconnections.com/content/3/1/R13.full.pdf+html

HOW FIBROBLAST GROWTH FACTOR 23 WORKS. Liu S1, Quarles LD. J Am Soc Nephrol. 2007 Jun;18(6):1637-47. Epub 2007 May 9.  http://www.ncbi.nlm.nih.gov/pubmed/17494882

While developed in the context of nutritional rickets, the following may have applicability to understanding the x-rays of XLH patients: RADIOGRAPHIC SCORING METHOD FOR THE ASSESSMENT OF THE SEVERITY OF NUTRITIONAL RICKETS, Tom D Thacher, P.R. Fischer, John Pettifor, Juliana Olufunke Lawson, b.J. Manaster, J.C. Reading. https://www.researchgate.net/publication/12425132_Radiographic_scoring_method_for_the_assessment_of_the_severity_of_nutritional_rickets


Recent journal articles:

CONVENTIONAL THERAPY IN ADULTS WITH XLH IMPROVES DENTAL MANIFESTATIONS, BUT NOT ENTHESOPATHY, Commentary by Econs MJ., J Clin Endocrinol Metab. 2015 Oct;100(10):3622-4. doi: 10.1210/jc.2015-3229. PMID: 26439151 http://www.ncbi.nlm.nih.gov/pubmed/26439151

CONVENTIONAL THERAPY IN ADULTS WITH X-LINKED HYPOPHOSPHATEMIA: EFFECTS ON ENTHESOPATHY AND DENTAL DISEASE.  Connor J, Olear EA, Insogna KL, Katz L, Baker S, Kaur R, Simpson CA, Sterpka J, Dubrow R, Zhang JH, Carpenter TO.  J Clin Endocrinol Metab. 2015 Oct;100(10):3625-32. doi: 10.1210/JC.2015-2199. Epub 2015 Jul 15. PMID: 26176801 http://www.ncbi.nlm.nih.gov/pubmed/26176801

Other materials:

Poster from ENDO2015 with results from a burden of illness study. Alison Skrinar, PhD, Ayla Marshall, Javier San Martin, MD, and Melita Dvorak-Ewell, PhD.  X-Linked Hypophosphatemia (XLH) Impairs Skeletal Health Outcomes and Physical Function in Affected Adults.  http://www.ultragenyx.com/file.cfm/22/docs/KRN23%20-%20ENDO%20adult%20disease%20burden%20poster%20March%202015.pdf

Poster from ENDO2015 with results from a burden of illness study. Agnes Linglart, MD, PhD , Melita Dvorak-Ewell, PhD , Ayla Marshall , Javier San Martin, MD , and Alison Skrinar, PhD.  Impaired Mobility and Pain Significantly Impact the Quality of Life of Children with X-Linked Hypophosphemia (XLH). http://www.ultragenyx.com/file.cfm/22/docs/KRN23%20-%20ICCBH%20pediatric%20disease%20burden%20poster%20June%202015.pdf