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Case Report

Individualized Dosage Optimization for Myeloablative Conditioning before Unrelated Cord Blood Transplantation in a Diamond–Blackfan Anemia Patient with Germline RPL11 Mutation: A Case Study

1
Department of Pediatric Hematology and Oncology, Koo Foundation Sun Yat-Sen Cancer Center, Taipei City 112, Taiwan
2
Department of Pharmacy, Koo Foundation Sun Yat-Sen Cancer Center, Taipei City 112, Taiwan
3
Institute of Molecular Biology, Academia Sinica, Taipei City 115, Taiwan
*
Authors to whom correspondence should be addressed.
Academic Editor: Alina Pyka-Pająk
Processes 2022, 10(2), 201; https://doi.org/10.3390/pr10020201
Received: 21 December 2021 / Revised: 17 January 2022 / Accepted: 18 January 2022 / Published: 21 January 2022
Unrelated cord blood transplantation (CBT) for Diamond–Blackfan anemia (DBA), a systemic ribosomopathy affecting the disposition of conditioning agents, has resulted in outcomes inferior to those by transplantations from matched donors. We report the experience of the pharmacokinetics-guided myeloablative unrelated CBT in a DBA patient with a germline RPL11 mutation. The conditioning consisted of individualized dosing of fludarabine (based on weight and renal function with a target area under the curve (AUC) of 17.5 mg·h/L) and busulfan (based on therapeutic drug monitoring with a target AUC of 90 mg·h/L), as well as dosing and timing of thymoglobulin (based on body weight and pre-dose lymphocyte count to target pre-CBT AUC of 30.7 AU·day/mL and post-CBT AUC of 4.3 AU·day/mL, respectively). The pharmacokinetic measures resulted in a 27.5% reduction in busulfan and a 35% increase in fludarabine, as well as an over three-fold increase in thymoglobulin dosage with the start time changed to day-9 instead of day-2 compared to regular regimens. The transplantation resulted in rapid, complete, and sustained hematopoietic engraftment. The patient is now healthy over 3 years after CBT. A pharmacokinetics-guided individualized dosing strategy for conditioning might be a feasible option to improve the outcomes of DBA patients receiving unrelated myeloablative CBT. View Full-Text
Keywords: population pharmacokinetics model; therapeutic drug monitoring; Diamond–Blackfan anemia; unrelated cord blood transplantation; myeloablative conditioning population pharmacokinetics model; therapeutic drug monitoring; Diamond–Blackfan anemia; unrelated cord blood transplantation; myeloablative conditioning
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MDPI and ACS Style

Chen, R.-L.; Fang, L.-H.; Chen, L.-Y. Individualized Dosage Optimization for Myeloablative Conditioning before Unrelated Cord Blood Transplantation in a Diamond–Blackfan Anemia Patient with Germline RPL11 Mutation: A Case Study. Processes 2022, 10, 201. https://doi.org/10.3390/pr10020201

AMA Style

Chen R-L, Fang L-H, Chen L-Y. Individualized Dosage Optimization for Myeloablative Conditioning before Unrelated Cord Blood Transplantation in a Diamond–Blackfan Anemia Patient with Germline RPL11 Mutation: A Case Study. Processes. 2022; 10(2):201. https://doi.org/10.3390/pr10020201

Chicago/Turabian Style

Chen, Rong-Long, Li-Hua Fang, and Liuh-Yow Chen. 2022. "Individualized Dosage Optimization for Myeloablative Conditioning before Unrelated Cord Blood Transplantation in a Diamond–Blackfan Anemia Patient with Germline RPL11 Mutation: A Case Study" Processes 10, no. 2: 201. https://doi.org/10.3390/pr10020201

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