Profile of asfotase alfa in the treatment of hypophosphatasia: design, development, and place in therapy

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Term Occurence Count Dictionary
hyperphosphatemia 1 endocrinologydiseases
hypophosphatasia 16 endocrinologydiseases
hypophosphatemia 1 endocrinologydiseases
prednisone 1 endocrinologydiseasesdrugs
Teriparatide 1 endocrinologydiseasesdrugs
calcinosis 9 endocrinologydiseases
hypercalcemia 8 endocrinologydiseases
osteoporosis 1 endocrinologydiseases
rickets 5 endocrinologydiseases
vascular calcification 1 endocrinologydiseases

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Select Drug Character Offset Drug Term Instance
Teriparatide 15847 formation and may disrupt mineralization, these drugs are strongly contraindicated for use in HPP patients. Teriparatide (TPTD), a recombinant peptide based on human PTH, is an anabolic agent used to treat osteoporosis. Administration
prednisone 13155 over time. Additional attempted interventions included administration of parathyroid hormone (PTH), prednisone , and normal plasma over several months; however, all of these approaches failed to make long-term improvements.[47]
Select Disease Character Offset Disease Term Instance
calcinosis 7416 mineral entry into the skeleton caused by elevated extracellular PPi levels, and hypercalciuria and nephro calcinosis can occur as consequences of hypercalcemia. Infants may also have muscle pain and weakness from a static
calcinosis 9473 in middle age, and commonly present with skeletal symptoms including fractures, osteomalacia, chondro calcinosis , osteoarthropathy, and stress fractures. Fractures can be at the hip or femoral neck, spine, or small
calcinosis 10018 loss. Musculoskeletal pain can be debilitating with restricted range of motion as a result of chondro calcinosis , sometimes leading to severe disability.Odontohypophosphatasia (odonto-HPP)While individuals diagnosed
calcinosis 28709 are not uniform. Below we highlight the response to treatment of neurological manifestations, nephro calcinosis , chronic pain, and craniosynostosis.Neurological manifestationsIn severe perinatal HPP, seizures resulting
calcinosis 30334 fine motor, and cognitive development as assessed by the Bayley Scales of Infant Development.[85]Nephro calcinosis Nephrocalcinosis can be observed in individuals with HPP at the time of diagnosis and is thought to be
calcinosis 30350 cognitive development as assessed by the Bayley Scales of Infant Development.[85]NephrocalcinosisNephro calcinosis can be observed in individuals with HPP at the time of diagnosis and is thought to be secondary to hypercalcemia
calcinosis 30583 hypercalciuria. This can cause impaired renal function. During clinical trials, no progression of nephro calcinosis was noted in patients with infantile HPP,[85],[87] and the status even improved in some patients.[85]
calcinosis 30716 with infantile HPP,[85],[87] and the status even improved in some patients.[85] Monitoring of nephro calcinosis by renal ultrasound is recommended at baseline and every 3 months in perinatal/infantile HPP and at
calcinosis 41360 ≥50 years, or Z-score ≤−2.0 in younger adult female and male patients with fractures or nephro calcinosis .[111] Ultimately, how AA will be employed in the long-term management of severely and mildly affected
hypercalcemia 7456 elevated extracellular PPi levels, and hypercalciuria and nephrocalcinosis can occur as consequences of hypercalcemia . Infants may also have muscle pain and weakness from a static myopathy, possibly as a result of an accumulation
hypercalcemia 7835 to intracranial hypertension.[31] Irritability and vomiting are common, which can be attributed to hypercalcemia or increased intracranial pressure with papilledema secondary to craniosynostosis. The infantile form
hypercalcemia 11938 insufficiency (ranging from oxygen supplementation with nasal cannula to mechanical ventilation), treatment of hypercalcemia with fluid hydration and low-calcium formula or diet, pain relief, and orthopedic surgeries for fractures.
hypercalcemia 12795 life-threatening infantile HPP.[45],[46] Treatment over ≥5 weeks elevated ALP into the normal range and improved hypercalcemia for all subjects and two subjects showed radiographic or histological improvement of skeletal manifestations.
hypercalcemia 30460 can be observed in individuals with HPP at the time of diagnosis and is thought to be secondary to hypercalcemia with hypercalciuria. This can cause impaired renal function. During clinical trials, no progression
hypercalcemia 34175 significantly with AA treatment.[85],[87],[93],[96]While calcium abnormalities are rare in adults with HPP, hypercalcemia is common in infants and children and often presents at diagnosis. Some infants continue to experience
hypercalcemia 34292 common in infants and children and often presents at diagnosis. Some infants continue to experience hypercalcemia and hyperphosphatemia after AA therapy, requiring continued use of low-calcium formula and/or a low-phosphorus
hypercalcemia 34963 large quantities of calcium for HAP deposition. Dietary calcium restriction should be liberalized once hypercalcemia is no longer present, or when serum PTH levels increase, to prevent hungry bones.[85] Serum PTH increases
hyperphosphatemia 34310 and children and often presents at diagnosis. Some infants continue to experience hypercalcemia and hyperphosphatemia after AA therapy, requiring continued use of low-calcium formula and/or a low-phosphorus formula.[87]
hypophosphatasia 88 Title: Drug Design, Development and TherapyProfile of asfotase alfa in the treatment of hypophosphatasia : design, development, and place in therapySasigarn A BowdenBrian L Foster1Division of Endocrinology,
hypophosphatasia 1873 therapy for individuals with HPP is discussed.Tissue-non-specific alkaline phosphatase (TNSALP) and hypophosphatasia (HPP)The enzyme, tissue-nonspecific alkaline phosphatase (TNSALP, TNALP, or TNAP), was first reported
hypophosphatasia 10075 restricted range of motion as a result of chondrocalcinosis, sometimes leading to severe disability.Odonto hypophosphatasia (odonto-HPP)While individuals diagnosed with odonto-HPP exhibit the biochemical characteristics of HPP,
hypophosphatasia 42768 Creative Commons Attribution 3.0 Unported License.Figure 2Skeletal and dental defects associated with hypophosphatasia .Notes: (A) MRI of the skull of a 6-year-old individual with hypophosphatasia (HPP) exhibiting craniosynostosis
hypophosphatasia 42845 defects associated with hypophosphatasia.Notes: (A) MRI of the skull of a 6-year-old individual with hypophosphatasia (HPP) exhibiting craniosynostosis and the resulting bregmatic bump. (B) Radiograph of a 4-year-old individual
hypophosphatasia 44261 Service Centre GmbH: Springer Nature [Child’s Nervous System]; Neurosurgical aspects of childhood hypophosphatasia , Collmann H, Mornet E, Gattenlöhner S, Beck C, Girschick H. Copyright 2009.[31] Images in E and F are
hypophosphatasia 44514 54(1), Berkseth KE, Tebben PJ, Drake MT, Hefferan TE, Jewison DE, Wermers RA, Clinical spectrum of hypophosphatasia diagnosed in adults, Pages 21–27, Copyright (2013), with permission from Elsevier.[36] Image in G
hypophosphatasia 44751 Reibel A, Manière MC, Clauss F, et al. Orodental phenotype and genotype findings in all subtypes of hypophosphatasia . Orphanet J Rare Dis. 2009;4(1):6. Image in H is reproduced with permission from © 2017 American Society
hypophosphatasia 45326 correlate: case study of identical twins with cementum and periodontal defects resulting from odonto hypophosphatasia . In: McCauley LK, Somerman MJ, eds. Mineralized Tissues in Oral and Craniofacial Science: Biological
hypophosphatasia 47110 Yadav MC, Lemire I, Leonard P, et al, Dose response of bone-targeted enzyme replacement for murine hypophosphatasia , Pages 250–256, Copyright (2011), with permission from Elsevier.[79] Images in panel C are reprinted
hypophosphatasia 47452 deficiency causes abnormal craniofacial bone development in the Alpl(−/−) mouse model of infantile hypophosphatasia , Pages 81–94, Copyright (2014), with permission from Elsevier.[71] Images in panel E are reproduced
hypophosphatasia 47758 Foster BL, Nagatomo KJ, Tso HW, et al. Tooth root dentin mineralization defects in a mouse model of hypophosphatasia . J Bone Miner Res. 2013;28(2):271–282.[70]Figure 4Asfotase alfa enzyme replacement therapy in a mouse
hypophosphatasia 49646 Copyright © 2008 ASBMR. Millán JL, Narisawa S, Lemire I, et al. Enzyme replacement therapy for murine hypophosphatasia . J Bone Miner Res. 2008;23(6):777–787.[76] Images in panel D are reprinted with permission from Bone,
hypophosphatasia 49896 Nam HK, et al, Enzyme replacement for craniofacial skeletal defects and craniosynostosis in murine hypophosphatasia , Pages 203–211, Copyright (2015) with permission from Elsevier.[80] H&E images in panel E are previously
hypophosphatasia 50184 replacement therapy in human subjects.Notes: Hand radiographs of a 15-year-old patient with severe childhood hypophosphatasia pre- and post-enzyme replacement therapy (ERT) treatment (6 months) with asfotase alfa. (A) Prior to
hypophosphatasia 50717 Osteoporosis International, Asfotase alfa treatment for 1 year in a 16 year-old male with severe childhood hypophosphatasia , Bowden SA, Adler BH, Copyright 2018.[93
hypophosphatemia 18453 (Montreal, Canada), focusing on another enzyme, PHEX, and its associated mineralization disorder, X-linked hypophosphatemia (XLH; OMIM# 307800).[74],[75] In order to engineer a soluble, secreted, and stable TNSALP that could
osteoporosis 15945 patients. Teriparatide (TPTD), a recombinant peptide based on human PTH, is an anabolic agent used to treat osteoporosis . Administration of TPTD to an adult with HPP and multiple stress fractures increased ALP levels, reduced
rickets 7226 of the chest that lead to respiratory insufficiency. Unlike other forms of genetic and nutritional rickets , serum calcium levels are generally high at diagnosis of HPP due to blocked mineral entry into the skeleton
rickets 8119 failure.Childhood HPPThis form features skeletal abnormalities that appear after 6 months, especially rickets manifesting as bowed legs and bony enlargement near joints due to widened metaphyses (Figure 2C and
rickets 13626 improved skeletal mineralization; however, the return of host hematopoiesis was accompanied by worsening rickets , scoliosis, and fractures by 6 months post-BMT.[48] A modified BMT approach employing marrow, implantation
rickets 13895 osteoblasts to a 9.5-month-old female with severe infantile HPP promoted slight improvements in severity of rickets , scoliosis, and skeletal mineralization, though minimal engraftment was evident.[49] A similar approach
rickets 24781 striking by 24 weeks of treatment. Specifically, radiographs revealed improved mineralization, healing of rickets , resolution of radiolucency and sclerosis, fracture healing, and reduced deformity.[85] The survival
vascular calcification 37524 recombinant enzyme, potential for adverse effects such as exacerbation of pathological calcification (eg, vascular calcification , kidney stones, eye calcifications; though these have not been documented to date), possibility of developing

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