Long-Term Outcomes of Early Enzyme Replacement Therapy With Asfotase Alfa in Perinatal Benign Hypophosphatasia: Amelioration of Bone Deformities in a Young Child.

Shuntaro Terayama, Masahisa Kobayashi, Tokumasa Suemitsu, Osamu Samura, Kimihiko Oishi

Abstract readCase Reports

In one paragraph

Article in Cureus, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it

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0citing papers in PubMed

–field-weighted citation impact

1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

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Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

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0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

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PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors.

Shuntaro TerayamaDepartment of Pediatrics, The Jikei University School of Medicine, Tokyo, JPN.

Masahisa KobayashiDepartment of Pediatrics, The Jikei University School of Medicine, Tokyo, JPN.

Tokumasa SuemitsuDepartment of Obstetrics and Gynecology, The Jikei University School of Medicine, Tokyo, JPN.

Osamu SamuraDepartment of Obstetrics and Gynecology, The Jikei University School of Medicine, Tokyo, JPN.

Kimihiko OishiDepartment of Pediatrics, The Jikei University School of Medicine, Tokyo, JPN.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hypophosphatasia (HPP) is a congenital skeletal dysplasia. Enzyme replacement therapy (ERT) improves survival and bone mineralization of patients with perinatal severe HPP. However, there are few reports of ERT in patients with perinatal benign HPP, and its long-term efficacy remains unclear. Herein, we report the case of a boy with perinatal benign HPP who was initiated on early ERT with asfotase alpha. The patient was suspected of having HPP because of shortened limbs and deformed long bones on fetal 3D-CT. He was diagnosed with HPP based on clinical manifestations, low serum alkaline phosphatase levels, and

Indexed as

asfotase alfabone deformitybone spurhypophosphatasiashort stature

Identifiers

PMID40051960

DOI10.7759/cureus.78473

PMCPMC11883447

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