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. 2025 Apr 28;25(1):787.
doi: 10.1186/s12885-025-14126-8.

Autologous stem cell transplantation for adults with Philadelphia-negative acute lymphoblastic leukemia in first complete remission. A study by the Acute Leukemia Working Party of the EBMT

Ryszard Swoboda  1 , Myriam Labopin  2   3 , Sebastian Giebel  4 , Johan Maertens  5 , Elena Parovichnikova  6 , Jurjen Versluis  7 , Jiri Pavlu  8 , Anna Kopinska  9 , Saveria Capria  10 , Ludek Raida  11 , Alessandro Rambaldi  12 , Denis Caillot  13 , Frantisek Folber  14 , David Nachbaur  15 , Mustafa Ozturk  16 , Mahmoud Aljurf  17 , Marie Thérèse Rubio  18 , Norbert Claude Gorin  19 , Francesco Lanza  20 , Arnon Nagler  21 , Mohamad Mohty  2   3 , Fabio Ciceri  22
Affiliations

Autologous stem cell transplantation for adults with Philadelphia-negative acute lymphoblastic leukemia in first complete remission. A study by the Acute Leukemia Working Party of the EBMT

Ryszard Swoboda et al. BMC Cancer. .

Abstract

Background: The role of autologous hematopoietic stem cell transplantation (AHSCT) in adults with Philadelphia chromosome-negative acute lymphoblastic leukemia (Ph-ALL) remains controversial. The aim of this retrospective study was to analyze results of AHSCT and to identify prognostic factors.

Methods: Overall, 700 patients transplanted in first complete remission between the years 1999-2020 were included. Median patient age was 31.9 years (68% male). B-cell precursor ALL (BCP-ALL) and T-cell precursor ALL (TCP-ALL) was diagnosed in 35% and 65%, respectively. Among 190 patients with available data, negative minimal residual disease (MRD) status was reported in 167 (88%) cases.

Results: The probabilities of overall survival (OS) and leukemia-free survival (LFS) at 2 years were 67% and 56%; relapse incidence (RI) and non-relapse mortality (NRM) were 39% and 5%, respectively. TCP-ALL was associated with lower RI (41% vs. 56%, p=0.001), higher LFS (52% vs. 38%, p=0.002) and OS (58% vs 45%, p=0.001) at 5 years when compared to BCP-ALL. In the multivariate analysis, TCP-ALL and longer interval from diagnosis do AHSCT were associated with reduced risk of relapse (HR 0.7, p=0.006 and HR=0.95, p=0.018), better LFS (HR=0.76, p=0.02 and HR=0.95, p=0.01) and OS (HR=0.75, p=0.024 and HR=0.94, p=0.013, respectively). Increasing patient age was associated with higher NRM (HR=1.49, p<0.0001), worse LFS (HR=1.1, p=0.01) and OS (HR=1.17, p=0.0001).

Conclusions: Autologous hematopoietic stem cell transplantation is relatively safe option of late treatment intensification in adults with Ph- ALL. It may be a valuable option especially in patients with TCP-ALL, however it should be proved in prospective clinical trials.

Keywords: Autologous stem cell transplantation; Complete remission; Philadelphia negative acute lymphoblastic leukemia.

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Conflict of interest statement

Declarations. Ethics approval and consent to participate: The study was approved by the ALWP of the EBMT institutional review board and conducted in accordance with the Declaration of Helsinki and Good Clinical Practice guidelines. Consent for publication: Not applicable. Competing interests: The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
Transplant outcomes in the whole population. NRM – non-relapse mortality; RI – relapse incidence; LFS – leukemia-free survival; OS – overall survival
Fig. 2
Fig. 2
Transplant outcomes in BCP- and TCP-ALL patients. NRM – non-relapse mortality; RI – relapse incidence; LFS – leukemia-free survival; OS – overall survival
See this image and copyright information in PMC

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