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Mapping PedsQL™ Generic Core Scales to EQ-5D-3L utility scores in transfusion-dependent thalassemia patients

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Abstract

Purpose

To develop a mapping algorithm for generating EQ-5D-3L utility scores from the PedsQL Generic Core Scales (PedsQL GCS) in patients with transfusion-dependent thalassemia (TDT).

Methods

The algorithm was developed using data from 345 TDT patients. Spearman’s rank correlation was used to evaluate the conceptual overlap between the instruments. Model specifications were chosen using a stepwise regression. Both direct and response mapping methods were attempted. Six mapping estimation methods ordinary least squares (OLS), a log-transformed response using OLS, generalized linear model (GLM), two-part model (TPM), Tobit and multinomial logistic regression (MLOGIT) were tested to determine the root mean squared error (RMSE) and mean absolute error (MAE). Other criterion used were accuracy of the predicted utility score, proportions of absolute differences that was less than 0.03 and intraclass correlation coefficient. An in-sample, leave-one-out cross validation was conducted to test the generalizability of each model.

Results

The best performing model was specified with three out of the four PedsQL GCS scales—the physical, emotional and social functioning score. The best performing estimation method for direct mapping was a GLM with a RMSE of 0.1273 and MAE of 0.1016, while the best estimation method for response mapping was the MLOGIT with a RMSE of 0.1597 and MAE of 0.0826.

Conclusion

The mapping algorithm developed using the GLM would facilitate the calculation of utility scores to inform economic evaluations for TDT patients when EQ-5D data is not available. However, caution should be exercised when using this algorithm in patients who have poor quality of life.

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Availability of data and material

The datasets used during the current study are available from the corresponding author on reasonable request.

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Acknowledgements

The authors would like to thank the Director General of the Ministry of Health Malaysia for the permission to publish the research findings of this study. We also would like to acknowledge the paediatricians, interviewers (Faizah Mohd Hanapiah, Mohd Hafizudin Mohd Salim, Mohamad Khudri Khairudin, Siti Nur Syahida Abdul Jalil, Muhammad Farid Abdul Ghaffar, Nur Liyana Kamaronzaman, Mohd Saharudin Mat Salim, Nurul Syafika Mohd Hairi, Analisa Coldelia Anak Rumpu, Elvyshirah Hadirin, Raphaela Romanus, Nur Asyilla Aslinda Mohd Nasir, Nurzuliana Hassim, Mohd Afiq Mohs Azis, Hamizah Kharber, Nur Athirah Abdul Razak, Nor Afifa Mazlan, Mohd Afiq Mohd Sulaiman, Nurain Syahirah Abu Bakar), patients, parents and caregivers from the various hospitals in Malaysia for participating and their cooperation in making this study possible. The authors would also like to thank Dr Annushiah Vasan Thakumar for her assistance and input during the analysis phase of the manuscript.

Funding

No financial support was received for this study.

Author information

Authors and Affiliations

  1. Discipline of Social and Administrative Pharmacy, School of Pharmaceutical Science, Universiti Sains Malaysia, 11800, Pulau Pinang, Malaysia

    Asrul Akmal Shafie, Irwinder Kaur Chhabra, Jacqueline Hui Yi Wong & Noor Syahireen Mohammed

  2. Institutional Planning and Strategic Centre, Universiti Sains Malaysia, 11800, Penang, Malaysia

    Asrul Akmal Shafie

  3. Pharmacy Department, Sabah Women and Children’s Hospital, Ministry of Health Malaysia, Karung Berkunci No.187, 88996, Kota Kinabalu, Sabah, Malaysia

    Irwinder Kaur Chhabra

  4. Pharmacy Department, Hospital Kuala Lumpur, Ministry of Health Malaysia, Jalan Pahang, 50586, Kuala Lumpur, Malaysia

    Jacqueline Hui Yi Wong

  5. Clinical Research Center, Hospital Sultanah Bahiyah, Ministry of Health Malaysia, KM 6, Jalan Langgar, 05460, Alor Setar, Kedah Darul Aman, Malaysia

    Noor Syahireen Mohammed

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  1. Asrul Akmal Shafie
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Contributions

AAS: was involved in the revision of the paper for intellectual content and in the design, interpretation, analysis of data and proofreading the manuscript; IKC: was involved in the drafting of the manuscript, analysis and interpretation of the data; JWHY: was involved in the design, collection, training, validation of the study and data and proofreading the manuscript; NSM: was involved in the training, validation of the study and data and proofreading the manuscript.

Corresponding author

Correspondence to Asrul Akmal Shafie.

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Conflict of interest

The authors declare that there is no conflict of interest.

Ethics approval

This study was registered with the Malaysian National Medical Research Registry (NMRR-17-2614-38966). This study was also approved by the Malaysian Research and Ethics Committee.

Consent to participate

Prior to each interview, written consents and assents were obtained from both caregivers and patients who were willing to participate.

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Shafie, A.A., Chhabra, I.K., Wong, J. et al. Mapping PedsQL™ Generic Core Scales to EQ-5D-3L utility scores in transfusion-dependent thalassemia patients. Eur J Health Econ 22, 735–747 (2021). https://doi.org/10.1007/s10198-021-01287-z

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