Quality-adjusted time without symptoms or toxicity analysis of haploidentical-related donor vs. identical sibling donor hematopoietic stem cell transplantation in acute myeloid leukemia

急性髓系白血病中单倍体相合亲缘供者与同胞相合供者造血干细胞移植的无症状或毒性质量调整时间分析

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Abstract

OBJECTIVE: 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). METHODS: 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=U(TOX) × TOX + U(TWiST) × TWiST + U(REL) × REL + U(aGVHD) × aGVHD + U(cGVHD) × cGVHD. RESULTS: 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: [Formula: see text][Formula: see text]. The model showed that when [Formula: see text], [Formula: see text], and [Formula: see text] were within the range of 0-1, as well as when [Formula: see text] 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., [Formula: see text]=0.5, [Formula: see text]=0.05, [Formula: see text]=0.5, and [Formula: see text]=0.5. The Q-TWiST values of ISD and HID HSCT were 896 and 900 d, respectively (P=0.470). CONCLUSIONS: We first observed that Q-TWiST was comparable between AML patients receiving HID HSCT and those receiving ISD HSCT.

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