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Tumoral calcinosis, hyperphosphatemic, familial, 3(HFTC3)

MedGen UID:
1638917
Concept ID:
C4693864
Disease or Syndrome
Synonyms: HFTC3; TUMORAL CALCINOSIS, FAMILIAL, HYPERPHOSPHATEMIC, 3; TUMORAL CALCINOSIS, HYPERPHOSPHATEMIC, FAMILIAL, 3
 
Gene (location): KL (13q13.1)
 
Monarch Initiative: MONDO:0060715
OMIM®: 617994

Definition

Hyperphosphatemic familial tumoral calcinosis (HFTC) is a rare autosomal recessive metabolic disorder characterized by the progressive deposition of basic calcium phosphate crystals in periarticular spaces, soft tissues, and sometimes bone (Chefetz et al., 2005). The biochemical hallmark of tumoral calcinosis is hyperphosphatemia caused by increased renal absorption of phosphate due to loss-of-function mutations in the FGF23 (605380) or GALNT3 (601756) gene. The term 'hyperostosis-hyperphosphatemia syndrome' (HHS) is sometimes used when the disorder is characterized by involvement of the long bones associated with the radiographic findings of periosteal reaction and cortical hyperostosis. Although some have distinguished HHS from FTC by the presence of bone involvement and the absence of skin involvement (Frishberg et al., 2005), Ichikawa et al. (2010) concluded that the 2 entities represent a continuous spectrum of the same disease, best described as familial hyperphosphatemic tumoral calcinosis. HFTC is considered to be the clinical converse of autosomal dominant hypophosphatemic rickets (ADHR; 193100), an allelic disorder caused by gain-of-function mutations in the FGF23 gene and associated with hypophosphatemia and decreased renal phosphate absorption (Chefetz et al., 2005; Ichikawa et al., 2005). For a general phenotypic description and a discussion of genetic heterogeneity of HFTC, see 211900. [from OMIM]

Additional description

From MedlinePlus Genetics
Hyperphosphatemic familial tumoral calcinosis (HFTC) is a condition characterized by an increase in the levels of phosphate in the blood (hyperphosphatemia) and abnormal deposits of phosphate and calcium (calcinosis) in the body's tissues. Calcinosis typically develops in early childhood to early adulthood, although in some people the deposits first appear in infancy or in late adulthood. Calcinosis usually occurs in and just under skin tissue around the joints, most often the hips, shoulders, and elbows. Calcinosis may also develop in the soft tissue of the feet, legs, and hands. Rarely, calcinosis occurs in blood vessels or in the brain and can cause serious health problems. The deposits develop over time and vary in size. Larger deposits form masses that are noticeable under the skin and can interfere with the function of joints and impair movement. These large deposits may appear tumor-like (tumoral), but they are not tumors or cancerous. The number and frequency of deposits varies among affected individuals; some develop few deposits during their lifetime, while others may develop many in a short period of time.

A similar condition called hyperphosphatemia-hyperostosis syndrome (HHS) results in increased levels of phosphate in the blood, excessive bone growth, and bone lesions. This condition used to be considered a separate disorder, but it is now thought to be a mild variant of HFTC.

Other features of HFTC include eye abnormalities such as calcium buildup in the clear front covering of the eye (corneal calcification) or angioid streaks that occur when tiny breaks form in the layer of tissue at the back of the eye called Bruch's membrane. Inflammation of the long bones (diaphysis) or excessive bone growth (hyperostosis) may occur. Some affected individuals have dental abnormalities. In males, small crystals of cholesterol can accumulate (microlithiasis) in the testicles, which usually causes no health problems.  https://medlineplus.gov/genetics/condition/hyperphosphatemic-familial-tumoral-calcinosis

Clinical features

From HPO
Fatigue
MedGen UID:
41971
Concept ID:
C0015672
Sign or Symptom
A subjective feeling of tiredness characterized by a lack of energy and motivation.
Polyuria
MedGen UID:
19404
Concept ID:
C0032617
Sign or Symptom
An increased rate of urine production.
Nephrolithiasis
MedGen UID:
98227
Concept ID:
C0392525
Disease or Syndrome
The presence of calculi (stones) in the kidneys.
Headache
MedGen UID:
9149
Concept ID:
C0018681
Sign or Symptom
Cephalgia, or pain sensed in various parts of the head, not confined to the area of distribution of any nerve.
Polydipsia
MedGen UID:
43214
Concept ID:
C0085602
Sign or Symptom
Excessive thirst manifested by excessive fluid intake.
Osteopenia
MedGen UID:
18222
Concept ID:
C0029453
Disease or Syndrome
Osteopenia is a term to define bone density that is not normal but also not as low as osteoporosis. By definition from the World Health Organization osteopenia is defined by bone densitometry as a T score -1 to -2.5.
Cerebral calcification
MedGen UID:
124360
Concept ID:
C0270685
Finding
The presence of calcium deposition within the cerebrum.
Metacarpal periosteal thickening
MedGen UID:
322396
Concept ID:
C1834347
Finding
Calvarial osteosclerosis
MedGen UID:
340927
Concept ID:
C1855657
Finding
An increase in bone density affecting the calvaria (roof of the skull).
Achilles tendon calcification
MedGen UID:
1375297
Concept ID:
C4476778
Anatomical Abnormality
Ectopic deposition of calcium salts in the Achilles tendon.
Hypercalcemia
MedGen UID:
5686
Concept ID:
C0020437
Disease or Syndrome
An abnormally increased calcium concentration in the blood.
Hyperphosphatemia
MedGen UID:
39326
Concept ID:
C0085681
Disease or Syndrome
An abnormally increased phosphate concentration in the blood.
Elevated circulating parathyroid hormone level
MedGen UID:
167805
Concept ID:
C0857973
Finding
An abnormal increased concentration of parathyroid hormone.
High serum calcitriol
MedGen UID:
1619023
Concept ID:
C4531136
Finding
An increased concentration of calcitriol in the blood. Calcitriol is also known as 1,25-dihydroxycholecalciferol or 1,25-dihydroxyvitamin D3.
Parathyroid hyperplasia
MedGen UID:
75767
Concept ID:
C0271844
Disease or Syndrome
Hyperplasia of the parathyroid gland.

Professional guidelines

PubMed

Boyce AM, Lee AE, Roszko KL, Gafni RI
Front Endocrinol (Lausanne) 2020;11:293. Epub 2020 May 8 doi: 10.3389/fendo.2020.00293. PMID: 32457699Free PMC Article
Nitschke Y, Rutsch F
Curr Osteoporos Rep 2017 Aug;15(4):255-270. doi: 10.1007/s11914-017-0370-3. PMID: 28585220

Recent clinical studies

Etiology

Zuo Q, Yang W, Liu B, Yan D, Wang Z, Wang H, Deng W, Cao X, Yang J
Front Endocrinol (Lausanne) 2022;13:1008800. Epub 2022 Sep 23 doi: 10.3389/fendo.2022.1008800. PMID: 36213261Free PMC Article
Chakhtoura M, Ramnitz MS, Khoury N, Nemer G, Shabb N, Abchee A, Berberi A, Hourani M, Collins M, Ichikawa S, El Hajj Fuleihan G
Osteoporos Int 2018 Sep;29(9):1987-2009. Epub 2018 Jun 20 doi: 10.1007/s00198-018-4574-x. PMID: 29923062
Nitschke Y, Rutsch F
Curr Osteoporos Rep 2017 Aug;15(4):255-270. doi: 10.1007/s11914-017-0370-3. PMID: 28585220
Ramnitz MS, Gourh P, Goldbach-Mansky R, Wodajo F, Ichikawa S, Econs MJ, White KE, Molinolo A, Chen MY, Heller T, Del Rivero J, Seo-Mayer P, Arabshahi B, Jackson MB, Hatab S, McCarthy E, Guthrie LC, Brillante BA, Gafni RI, Collins MT
J Bone Miner Res 2016 Oct;31(10):1845-1854. Epub 2016 Sep 20 doi: 10.1002/jbmr.2870. PMID: 27164190Free PMC Article
Jost J, Bahans C, Courbebaisse M, Tran TA, Linglart A, Benistan K, Lienhardt A, Mutar H, Pfender E, Ratsimbazafy V, Guigonis V
J Clin Endocrinol Metab 2016 Jul;101(7):2810-5. Epub 2016 May 10 doi: 10.1210/jc.2016-1087. PMID: 27163355

Diagnosis

Moran H, Malvar M, Yuksel S, Bleich D
BMJ Case Rep 2024 Oct 11;17(10) doi: 10.1136/bcr-2023-259455. PMID: 39395830
Pallone SG, Kunii IS, da Silva REC, Lazaretti-Castro M
Calcif Tissue Int 2022 Jul;111(1):102-106. Epub 2022 Mar 25 doi: 10.1007/s00223-022-00969-x. PMID: 35338393
Kışla Ekinci RM, Gürbüz F, Balcı S, Bişgin A, Taştan M, Yüksel B, Yılmaz M
J Clin Res Pediatr Endocrinol 2019 Feb 20;11(1):94-99. Epub 2018 Jul 17 doi: 10.4274/jcrpe.galenos.2018.2018.0134. PMID: 30015621Free PMC Article
Jost J, Bahans C, Courbebaisse M, Tran TA, Linglart A, Benistan K, Lienhardt A, Mutar H, Pfender E, Ratsimbazafy V, Guigonis V
J Clin Endocrinol Metab 2016 Jul;101(7):2810-5. Epub 2016 May 10 doi: 10.1210/jc.2016-1087. PMID: 27163355
Rafaelsen S, Johansson S, Ræder H, Bjerknes R
BMC Genet 2014 Sep 24;15:98. doi: 10.1186/s12863-014-0098-3. PMID: 25249269Free PMC Article

Therapy

Fabbriciani G, Colombini A, Messina C, Adami G
Reumatismo 2024 Jun 24;76(2) doi: 10.4081/reumatismo.2024.1687. PMID: 38916164
Pallone SG, Kunii IS, da Silva REC, Lazaretti-Castro M
Calcif Tissue Int 2022 Jul;111(1):102-106. Epub 2022 Mar 25 doi: 10.1007/s00223-022-00969-x. PMID: 35338393
Ramnitz MS, Gourh P, Goldbach-Mansky R, Wodajo F, Ichikawa S, Econs MJ, White KE, Molinolo A, Chen MY, Heller T, Del Rivero J, Seo-Mayer P, Arabshahi B, Jackson MB, Hatab S, McCarthy E, Guthrie LC, Brillante BA, Gafni RI, Collins MT
J Bone Miner Res 2016 Oct;31(10):1845-1854. Epub 2016 Sep 20 doi: 10.1002/jbmr.2870. PMID: 27164190Free PMC Article
Jost J, Bahans C, Courbebaisse M, Tran TA, Linglart A, Benistan K, Lienhardt A, Mutar H, Pfender E, Ratsimbazafy V, Guigonis V
J Clin Endocrinol Metab 2016 Jul;101(7):2810-5. Epub 2016 May 10 doi: 10.1210/jc.2016-1087. PMID: 27163355
Finer G, Price HE, Shore RM, White KE, Langman CB
Am J Med Genet A 2014 Jun;164A(6):1545-9. Epub 2014 Mar 25 doi: 10.1002/ajmg.a.36476. PMID: 24668887

Prognosis

Jost J, Bahans C, Courbebaisse M, Tran TA, Linglart A, Benistan K, Lienhardt A, Mutar H, Pfender E, Ratsimbazafy V, Guigonis V
J Clin Endocrinol Metab 2016 Jul;101(7):2810-5. Epub 2016 May 10 doi: 10.1210/jc.2016-1087. PMID: 27163355
Rafaelsen S, Johansson S, Ræder H, Bjerknes R
BMC Genet 2014 Sep 24;15:98. doi: 10.1186/s12863-014-0098-3. PMID: 25249269Free PMC Article
Finer G, Price HE, Shore RM, White KE, Langman CB
Am J Med Genet A 2014 Jun;164A(6):1545-9. Epub 2014 Mar 25 doi: 10.1002/ajmg.a.36476. PMID: 24668887

Clinical prediction guides

Fabbriciani G, Colombini A, Messina C, Adami G
Reumatismo 2024 Jun 24;76(2) doi: 10.4081/reumatismo.2024.1687. PMID: 38916164
Ramnitz MS, Gourh P, Goldbach-Mansky R, Wodajo F, Ichikawa S, Econs MJ, White KE, Molinolo A, Chen MY, Heller T, Del Rivero J, Seo-Mayer P, Arabshahi B, Jackson MB, Hatab S, McCarthy E, Guthrie LC, Brillante BA, Gafni RI, Collins MT
J Bone Miner Res 2016 Oct;31(10):1845-1854. Epub 2016 Sep 20 doi: 10.1002/jbmr.2870. PMID: 27164190Free PMC Article
Jost J, Bahans C, Courbebaisse M, Tran TA, Linglart A, Benistan K, Lienhardt A, Mutar H, Pfender E, Ratsimbazafy V, Guigonis V
J Clin Endocrinol Metab 2016 Jul;101(7):2810-5. Epub 2016 May 10 doi: 10.1210/jc.2016-1087. PMID: 27163355

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