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Record W4405416708 · doi:10.1177/20406207241298088

Demographics, clinical characteristics, and real-world treatment patterns among patients with beta-thalassemia: a retrospective medical record abstraction study

2024· article· en· W4405416708 on OpenAlexaboutno aff
Maria Domenica Cappellini, Mrudula B. Glassberg, Juliana Meyers, Maria Jimenez, Tram Nham, Luciana Bueno, Jan Sieluk, Aylin Yücel, Ferras Alashkar

Bibliographic record

VenueTherapeutic Advances in Hematology · 2024
Typearticle
Languageen
FieldMedicine
TopicHemoglobinopathies and Related Disorders
Canadian institutionsnot available
FundersAgios PharmaceuticalsVertex PharmaceuticalsSanofiPfizerBristol-Myers Squibb
KeywordsMedicineBlood transfusionThalassemiaInternal medicineRetrospective cohort studyDemographicsAnemiaHemoglobinMedical recordPediatricsGastroenterologySurgeryDemography

Abstract

fetched live from OpenAlex

Background: Beta-thalassemias (BTs) are characterized by deficient or absent synthesis of the beta-globin subunit, leading to anemia. Patient characteristics and treatment patterns in these patients may vary. Objective: This retrospective study evaluated demographics, clinical characteristics, and treatment patterns in patients with transfusion-dependent BT (TDT) and non-transfusion-dependent BT (NTDT). Methods: Medical records of adults with TDT or NTDT in the United Kingdom, France, Germany, Spain, and Canada with ⩾5 years of history within the practice were evaluated. Results: = 96), mean (SD) age was 36.6 (9.8) years, and 38.5% were female. Among patients with TDT, 21.2% received transfusions every 2 weeks or more frequently, 28.8% every 3 weeks, 26.3% every 4 weeks, and 21.2% less frequently than 4 weeks. Patients with TDT had a mean (SD) of 2.4 (0.6) units of blood transfused per transfusion, with a pretransfusion hemoglobin (Hb) level of 6.9 (1.3). In total, 84.4% of patients with NTDT had at least one transfusion, and the mean (SD) number of transfusions among patients with NTDT was 15.9 (15.9). Among patients with NTDT, the mean (SD) units of blood per transfusion were 2.2 (0.6) units, and the mean (SD) Hb level prior to transfusion was 7.4 (1.2) g/dL. Iron chelation therapy was received by 70.3% of TDT patients and 45.8% of NTDT patients. Conclusion: This study found that both patients with NTDT and TDT have low pretransfusion Hb levels. A high number of patients, especially patients with TDT, were not treated according to the current recommendations on target hemoglobin level, thereby highlighting the importance of national reference centers for improving long-term outcomes and quality of life in these patients.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.106
Threshold uncertainty score0.740

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.001
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.015
GPT teacher head0.336
Teacher spread0.322 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designObservational
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations2
Published2024
Admission routes1
Has abstractyes

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