Literature DB >> 33549117

A bedridden young lady with hypophosphatemic rickets treated with denosumab: a case report.

Butheinah A Al-Sharafi1, Nuha A Al-Yousfi2, Said A Bamashmus3.   

Abstract

BACKGROUND: Hypophosphatemic rickets is associated with delayed walking, bone deformities, growth failure and physical dysfunction that can limit daily activities. Treatment consists of phosphate salts and calcitriol. We report a case that received denosumab with marked improvement in her condition. CASE
PRESENTATION: A 24-year-old Yemeni female with hypophosphatemic rickets presented to an endocrinologist with severe weakness and severe pain in the extremities, she had been bedridden for the last 4 years. Bone density showed severe osteoporosis (T score of hip was - 5.0 and Z score of hip was - 5.0, T score of the spine was - 6.0 and Z score of the spine was - 6.1) so the patient was started on denosumab in addition to calcitriol and after 7months she was feeling stronger and felt she could stand assisted and was walking with assistance within 9 months and after 1.5 years of treatment she was walking unassisted.
CONCLUSION: Denosumab is an effective treatment for osteoporosis, we used it in our patient in addition to calcitriol because she had severe osteoporosis due to long standing hypophosphatemic rickets that had not been treated properly, the patient improved markedly and regained the ability to walk again after being bedridden for 4 years. It may be a drug to consider in such cases although further studies need to be done to confirm this.

Entities:  

Keywords:  Case report; Denosumab; Hyperparathyroidism; Hypophosphatemic rickets; Osteomalacia; Osteoporosis

Mesh:

Substances:

Year:  2021        PMID: 33549117      PMCID: PMC7868015          DOI: 10.1186/s13256-020-02654-9

Source DB:  PubMed          Journal:  J Med Case Rep        ISSN: 1752-1947


  11 in total

1.  Burosumab Therapy in Children with X-Linked Hypophosphatemia.

Authors:  Thomas O Carpenter; Michael P Whyte; Erik A Imel; Annemieke M Boot; Wolfgang Högler; Agnès Linglart; Raja Padidela; William Van't Hoff; Meng Mao; Chao-Yin Chen; Alison Skrinar; Emil Kakkis; Javier San Martin; Anthony A Portale
Journal:  N Engl J Med       Date:  2018-05-24       Impact factor: 91.245

Review 2.  Current and Emerging Therapeutic Options for the Management of Rare Skeletal Diseases.

Authors:  Oliver Semler; Mirko Rehberg; Nava Mehdiani; Miriam Jackels; Heike Hoyer-Kuhn
Journal:  Paediatr Drugs       Date:  2019-04       Impact factor: 3.022

Review 3.  Osteocyte regulation of phosphate homeostasis and bone mineralization underlies the pathophysiology of the heritable disorders of rickets and osteomalacia.

Authors:  Jian Q Feng; Erica L Clinkenbeard; Baozhi Yuan; Kenneth E White; Marc K Drezner
Journal:  Bone       Date:  2013-02-09       Impact factor: 4.398

4.  A prospective trial of phosphate and 1,25-dihydroxyvitamin D3 therapy in symptomatic adults with X-linked hypophosphatemic rickets.

Authors:  W Sullivan; T Carpenter; F Glorieux; R Travers; K Insogna
Journal:  J Clin Endocrinol Metab       Date:  1992-09       Impact factor: 5.958

5.  A translocation causing increased alpha-klotho level results in hypophosphatemic rickets and hyperparathyroidism.

Authors:  Catherine A Brownstein; Felix Adler; Carol Nelson-Williams; Junko Iijima; Peining Li; Akihiro Imura; Yo-Ichi Nabeshima; Miguel Reyes-Mugica; Thomas O Carpenter; Richard P Lifton
Journal:  Proc Natl Acad Sci U S A       Date:  2008-02-28       Impact factor: 11.205

6.  Denosumab improves clinical manifestations of hypophosphatemic osteomalacia by adefovir-induced Fanconi syndrome: a case report.

Authors:  Tomohisa Kunii; Toshie Iijima; Teruo Jojima; Masanori Shimizu; Masato Kase; Shintaro Sakurai; Takahiko Kogai; Isao Usui; Yoshimasa Aso
Journal:  J Med Case Rep       Date:  2019-04-20

7.  X-linked hypophosphatemic rickets: an Italian experts' opinion survey.

Authors:  F Emma; M Cappa; F Antoniazzi; M L Bianchi; I Chiodini; C Eller Vainicher; N Di Iorgi; M Maghnie; A Cassio; A Balsamo; F Baronio; L de Sanctis; D Tessaris; G I Baroncelli; S Mora; M L Brandi; G Weber; A D'Ausilio; E P Lanati
Journal:  Ital J Pediatr       Date:  2019-05-31       Impact factor: 2.638

8.  Individualized treatment with denosumab in children with osteogenesis imperfecta - follow up of a trial cohort.

Authors:  Heike Hoyer-Kuhn; Mirko Rehberg; Christian Netzer; Eckhard Schoenau; Oliver Semler
Journal:  Orphanet J Rare Dis       Date:  2019-09-18       Impact factor: 4.123

9.  Use of Denosumab in Children With Osteoclast Bone Dysplasias: Report of Three Cases.

Authors:  Alexander Upfill-Brown; Susan Bukata; Nicholas M Bernthal; Alan L Felsenfeld; Scott D Nelson; Arun Singh; Katherine Wesseling-Perry; Fritz C Eilber; Noah C Federman
Journal:  JBMR Plus       Date:  2019-08-22

10.  Therapeutic management of hypophosphatemic rickets from infancy to adulthood.

Authors:  Agnès Linglart; Martin Biosse-Duplan; Karine Briot; Catherine Chaussain; Laure Esterle; Séverine Guillaume-Czitrom; Peter Kamenicky; Jerome Nevoux; Dominique Prié; Anya Rothenbuhler; Philippe Wicart; Pol Harvengt
Journal:  Endocr Connect       Date:  2014-03-14       Impact factor: 3.335

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