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A Delphi panel to build consensus on assessing disease severity and disease progression in adult patients with hypophosphatasia in the United States.
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- Author(s): Dahir KM;Dahir KM; Rush ET; Rush ET; Rush ET; Rush ET; Diaz-Mendoza S; Diaz-Mendoza S; Kishnani PS; Kishnani PS
- Source:
Journal of endocrinological investigation [J Endocrinol Invest] 2024 Jun; Vol. 47 (6), pp. 1487-1497. Date of Electronic Publication: 2024 Jan 18.- Publication Type:
Journal Article- Language:
English - Source:
- Additional Information
- Source: Publisher: Springer Country of Publication: Italy NLM ID: 7806594 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1720-8386 (Electronic) Linking ISSN: 03914097 NLM ISO Abbreviation: J Endocrinol Invest Subsets: MEDLINE
- Publication Information: Publication: 2014- : Berlin : Springer
Original Publication: Milano, Published for the Italian Society of Endocrinology by Editrice Kurtis. - Subject Terms:
- Abstract: Background: Hypophosphatasia (HPP) is an inborn error of metabolism with a variable presentation. We conducted a modified Delphi panel to obtain expert consensus on knowledge gaps regarding disease severity and progression in adult patients with HPP.
Methods: Healthcare professionals (HCPs) with experience managing adult patients with HPP were recruited to participate in a 3-round Delphi panel (round 1: paper survey and 1:1 interview; rounds 2-3: email survey). Panelists rated the extent of their agreement with statements about disease severity and progression in adult patients with HPP. Consensus was defined as ≥ 80% agreement.
Results: Ten HCPs completed round 1; nine completed rounds 2 and 3. Consensus was reached on 46/120 statements derived from steering committee input. Disease severity markers in adult patients with HPP can be bone-related (recurrent/poorly healing fractures, pseudo-fractures, metatarsal fractures, osteomalacia) or involve dentition or physiologic/functional manifestations (use of mobility devices/home modifications, abnormal gait, pain). Disease progression markers can include recurrent/poorly healing low-trauma fractures, development of ectopic calcifications, and/or impairment of functional activity. Panelists supported the development of a tool to help assess disease severity in the clinic and track changes in severity over time. Panelists also highlighted the role of a multidisciplinary team, centers with expertise, and the need to refer patients when disease severity is not clear.
Conclusions: These statements regarding disease severity, progression, and assessment methods address some knowledge gaps in adult patients with HPP and may be helpful for treating HCPs, although the small sample size affects the ability to generalize the healthcare provider experience.
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- Publication Date: Date Created: 20240118 Date Completed: 20240601 Latest Revision: 20240603
- Publication Date: 20240603
- Accession Number: PMC11142979
- Accession Number: 10.1007/s40618-023-02256-4
- Accession Number: 38236379
- Source:
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