Quality-adjusted time without symptoms or toxicity analysis of haploidentical-related donor vs. identical sibling donor hematopoietic stem cell transplantation in acute myeloid leukemia
Yuewen Wang, Xianli Gao, Ting Wang, Xiaohui Zhang, Lan-Ping Xu, Yu Wang, Chen-Hua Yan, Huan Chen, Yuhong Chen, Wei Han, Feng‐Rong Wang, Jingzhi Wang, Xia Yan, Xiao‐Dong Mo
Abstract
<sec><b>Objective</b>We aimed to compare the quality-adjusted time without symptoms or toxicity (Q-TWiST) in acute myeloid leukemia (AML) patients who received haploidentical-related donor (HID) and identical sibling donor (ISD) hematopoietic stem cell transplantation (HSCT). </sec><sec><b>Methods</b>Five clinical health states were defined: toxicity (TOX), acute graft-versus-host disease (GVHD), chronic GVHD (cGVHD), time without symptoms and toxicity (TWiST) and relapse (REL). The equation used in this study was as follows: Q-TWiST=<i>U<sub>TOX</sub></i> × TOX + <i>U<sub>TWiST</sub></i> × TWiST + <i>U<sub>REL</sub></i> × REL + <i>U<sub>aGVHD</sub></i> × aGVHD + <i>U<sub>cGVHD</sub></i> × cGVHD. </sec><sec><b>Results</b>A total of 239 AML patients were enrolled. We established a mathematical model, i.e., Q-TWiST HID HSCT > Q-TWiST ISD HSCT, to explore the range of utility coefficients satisfying the inequality. Based on the raw data, the utility coefficient is equivalent to the following inequality: <inline-formula><tex-math id="M7">\begin{document}$ 10.57067{U}_{TOX}-46.27733{U}_{REL}+ $\end{document}</tex-math></inline-formula><inline-formula><tex-math id="M7-1">\begin{document}$ 105.9374+3.388078{U}_{aGVHD}-210.8198{U}_{cGVHD} > 0 $\end{document}</tex-math></inline-formula>. The model showed that when <inline-formula><tex-math id="M8">\begin{document}$ {U}_{TOX} $\end{document}</tex-math></inline-formula>, <inline-formula><tex-math id="M9">\begin{document}$ {U}_{REL} $\end{document}</tex-math></inline-formula>, and <inline-formula><tex-math id="M10">\begin{document}$ {U}_{aGVHD} $\end{document}</tex-math></inline-formula> were within the range of 0−1, as well as when <inline-formula><tex-math id="M11">\begin{document}$ {U}_{cGVHD} $\end{document}</tex-math></inline-formula> was within the range of 0−0.569, the inequality Q-TWiST HID HSCT > Q-TWiST ISD HSCT was valid. According to the results of the ChiCTR1800016972 study, the median coefficients of TOX, acute GVHD (aGVHD), and cGVHD were 0.56 (0.41−0.76), 0.56 (0.47−0.72), and 0.54 (0.37−0.79), respectively. We selected a series of specific examples of the coefficients, i.e., <inline-formula><tex-math id="M12">\begin{document}$ {U}_{TOX} $\end{document}</tex-math></inline-formula>=0.5, <inline-formula><tex-math id="M13">\begin{document}$ {U}_{REL} $\end{document}</tex-math></inline-formula>=0.05, <inline-formula><tex-math id="M14">\begin{document}$ {U}_{aGVHD} $\end{document}</tex-math></inline-formula>=0.5, and <inline-formula><tex-math id="M15">\begin{document}$ {U}_{cGVHD} $\end{document}</tex-math></inline-formula>=0.5. The Q-TWiST values of ISD and HID HSCT were 896 and 900 d, respectively (P=0.470). </sec><sec><b>Conclusions</b>We first observed that Q-TWiST was comparable between AML patients receiving HID HSCT and those receiving ISD HSCT.</sec>