Oral versus intravenous melphalan for autologous stem cell transplantation conditioning in lymphoma and multiple myeloma: a retrospective cohort study
- 1Department of Hematology, Shanxi Province Cancer Hospital/Shanxi Hospital Affiliated to Cancer Hospital, Chinese Academy of Medical Sciences/Cancer Hospital Affiliated to Shanxi Medical University
- 2Department of Pharmacy, Shanxi Province Cancer Hospital/Shanxi Hospital Affiliated to Cancer Hospital, Chinese Academy of Medical Sciences/Cancer Hospital Affiliated to Shanxi Medical University
Abstract
Objective: To compare oral (PO) and intravenous (IV) melphalan as conditioning regimens for autologous stem cell transplantation in patients with lymphoma or multiple myeloma (MM).
Methods: A retrospective propensity score-matched cohort analysis of 106 patients (76 with lymphoma and 30 with MM) was conducted to compare engraftment outcomes, adverse events, and survival between PO and IV melphalan conditioning. The primary endpoint was 3-year progression-free survival.
Results: The BEAM (carmustine, etoposide, cytarabine, and melphalan) regimen is the standard conditioning regimen for lymphoma and a single-agent melphalan was administered for MM. In patients with lymphoma, IV melphalan was associated with faster neutrophil engraftment than PO melphalan (10 vs 12 days, p = 0.025). In MM, IV melphalan was associated with faster platelet (PLT) engraftment (11 vs 14 days, p = 0.021). The PO group had higher grade 3–4 anemia (37.74% vs 18.87%, p = 0.031) but lower febrile neutropenia (35.85% vs 56.60%, p = 0.032) and mucositis (7.55% vs 33.96%, p = 0.001). In the PO group, grade 3–4 anemia correlated with delayed PLT engraftment (62.5% vs 21.4%, p = 0.008). Compared with IV melphalan, divided-dose oral melphalan was associated with lower rates of diarrhea (18.87% vs 37.74%) and oral mucositis (7.55% vs 33.96%), with an overall gastrointestinal toxicity rate (defined as at least one gastrointestinal adverse event) of 21.7% in the PO group versus 56.60% in the IV group. Complete response rates and 3-year survival outcomes were comparable between the groups.
Conclusion: Oral melphalan is a pragmatic alternative when IV melphalan is unavailable, with comparable efficacy and a distinct toxicity profile. Given variable bioavailability, it should not be considered bioequivalent to IV melphalan. Prospective pharmacokinetic studies are warranted to validate these findings.
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BibTeX
@article{wang2026oral,
title = {{Oral versus intravenous melphalan for autologous stem cell transplantation conditioning in lymphoma and multiple myeloma: a retrospective cohort study}},
author = {Jingrong Wang and Yimeng Guo and Jin Zhao and Xiaolian Wen and Liping Su and Zhiying Hao and Jing Yang},
journal = {Frontiers in Oncology},
year = {2026},
month = oct,
doi = {10.3389/fonc.2026.1923679},
url = {https://www.frontiersin.org/articles/10.3389/fonc.2026.1923679/full},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Oral versus intravenous melphalan for autologous stem cell transplantation conditioning in lymphoma and multiple myeloma: a retrospective cohort study
AU - Jingrong Wang
AU - Yimeng Guo
AU - Jin Zhao
AU - Xiaolian Wen
AU - Liping Su
AU - Zhiying Hao
AU - Jing Yang
PY - 2026
DA - 2026/10/19
JO - Frontiers in Oncology
DO - 10.3389/fonc.2026.1923679
UR - https://www.frontiersin.org/articles/10.3389/fonc.2026.1923679/full
AB - Objective: To compare oral (PO) and intravenous (IV) melphalan as conditioning regimens for autologous stem cell transplantation in patients with lymphoma or multiple myeloma (MM). Methods: A retrospective propensity score-matched cohort analysis of 106 patients (76 with lymphoma and 30 with MM) was conducted to compare engraftment outcomes, adverse events, and survival between PO and IV melphalan conditioning. The primary endpoint was 3-year progression-free survival. Results: The BEAM (carmustine, etoposide, cytarabine, and melphalan) regimen is the standard conditioning regimen for lymphoma and a single-agent melphalan was administered for MM. In patients with lymphoma, IV melphalan was associated with faster neutrophil engraftment than PO melphalan (10 vs 12 days, p = 0.025). In MM, IV melphalan was associated with faster platelet (PLT) engraftment (11 vs 14 days, p = 0.021). The PO group had higher grade 3–4 anemia (37.74% vs 18.87%, p = 0.031) but lower febrile neutropenia (35.85% vs 56.60%, p = 0.032) and mucositis (7.55% vs 33.96%, p = 0.001). In the PO group, grade 3–4 anemia correlated with delayed PLT engraftment (62.5% vs 21.4%, p = 0.008). Compared with IV melphalan, divided-dose oral melphalan was associated with lower rates of diarrhea (18.87% vs 37.74%) and oral mucositis (7.55% vs 33.96%), with an overall gastrointestinal toxicity rate (defined as at least one gastrointestinal adverse event) of 21.7% in the PO group versus 56.60% in the IV group. Complete response rates and 3-year survival outcomes were comparable between the groups. Conclusion: Oral melphalan is a pragmatic alternative when IV melphalan is unavailable, with comparable efficacy and a distinct toxicity profile. Given variable bioavailability, it should not be considered bioequivalent to IV melphalan. Prospective pharmacokinetic studies are warranted to validate these findings.
SN - 2234-943X
ER -