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Peer-reviewedCC BY 4.0Cancers

Long-Term Survival of Patients with Relapsed/Refractory Acute Leukemia Receiving Clofarabine/Cyclophosphamide Prior to Hematopoietic Stem Cell Transplantation

  • Irene Graf1,2
  • Philipp Wohlfarth3
  • Michael Kundi4
  • Margit Mitterbauer3
  • Axel Schulenburg3
  • Alexander W. Hauswirth1
  • Karoline V. Gleixner1,2
  • Hanna A. Knaus3
  • Lina Z. Rüsing3
  • Werner Rabitsch3
  • Peter Valent1,2
  • Wolfgang R. Sperr1,2
4 affiliations
  1. 1Division of Hematology and Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, 1090 Vienna, Austria
  2. 2Ludwig Boltzmann Institute for Hematology and Oncology, Medical University of Vienna, 1090 Vienna, Austria
  3. 3Bone Marrow Transplantation Unit, Department of Internal Medicine I, Medical University of Vienna, 1090 Vienna, Austria
  4. 4Center for Public Health, Department of Environmental Health, Medical University of Vienna, 1090 Vienna, Austria
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Abstract

Background: Allogeneic hematopoietic stem cell transplantation (HSCT) is the only potentially curative option for patients with drug-refractory acute myeloid (AML) or acute lymphoblastic leukemia (ALL). Patients and Methods: We retrospectively analyzed 82 patients (median age 47.5 years, range 19–69; female:male ratio 1:1.28) with drug-refractory (R) or relapsed/refractory (RR) acute leukemia (AML n = 66; ALL n = 16) who were treated with clofarabine (10 mg/m2) and cyclophosphamide (200 mg/m2) days 1–4 (ClofCy) prior to HSCT. Results: ClofCy resulted in rapid cytoreduction (median time to granulocytes <0.5 × 109/L: 5 days). HSCT was performed in 62.2% (51/82) of our patients. Infections (18/31, 58%) were the main reason to withhold transplantation. HSCT and non-HSCT patients differed significantly regarding the median number of chemotherapies received prior to ClofCy (HSCT, median = two therapies; non-HSCT, median = three therapies; p = 0.027). Median overall survival (OS) was 0.31 years in all patients (5- and 10-year OS; 24% and 20%, respectively). HSCT patients had a median OS of 1.23 years (5- and 10-year OS; 38.4% and 31%, respectively) and those in the non-HSCT cohort a median OS of 0.11 years (5-year OS; 0%). Median OS and relapse-free survival (RFS) in the HSCT patient cohort were 1.04 and 0.9 years, respectively. Acute graft-versus-host disease (aGvHD) was associated with inferior survival (median OS 0.46 years), whereas chronic graft-versus-host disease (cGvHD) was associated with improved OS and RFS (above 50%). Conclusion: ClofCy is an effective bridging regimen enabling HSCT in patients with R/RR acute leukemia. Introducing ClofCy may improve the probability of reaching HSCT and long-term survival in this cohort of patients with poor prognosis.

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Cite

BibTeXRIS
BibTeX
@article{graf2026longterm,
  title = {{Long-Term Survival of Patients with Relapsed/Refractory Acute Leukemia Receiving Clofarabine/Cyclophosphamide Prior to Hematopoietic Stem Cell Transplantation}},
  author = {Irene Graf and Philipp Wohlfarth and Michael Kundi and Margit Mitterbauer and Axel Schulenburg and Alexander W. Hauswirth and Karoline V. Gleixner and Hanna A. Knaus and Lina Z. Rüsing and Werner Rabitsch and Peter Valent and Wolfgang R. Sperr},
  journal = {Cancers},
  year = {2026},
  month = nov,
  doi = {10.3390/cancers18213593},
  url = {https://www.mdpi.com/2072-6694/18/21/3593},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Long-Term Survival of Patients with Relapsed/Refractory Acute Leukemia Receiving Clofarabine/Cyclophosphamide Prior to Hematopoietic Stem Cell Transplantation
AU  - Irene Graf
AU  - Philipp Wohlfarth
AU  - Michael Kundi
AU  - Margit Mitterbauer
AU  - Axel Schulenburg
AU  - Alexander W. Hauswirth
AU  - Karoline V. Gleixner
AU  - Hanna A. Knaus
AU  - Lina Z. Rüsing
AU  - Werner Rabitsch
AU  - Peter Valent
AU  - Wolfgang R. Sperr
PY  - 2026
DA  - 2026/11/09
JO  - Cancers
DO  - 10.3390/cancers18213593
UR  - https://www.mdpi.com/2072-6694/18/21/3593
AB  - Background: Allogeneic hematopoietic stem cell transplantation (HSCT) is the only potentially curative option for patients with drug-refractory acute myeloid (AML) or acute lymphoblastic leukemia (ALL). Patients and Methods: We retrospectively analyzed 82 patients (median age 47.5 years, range 19–69; female:male ratio 1:1.28) with drug-refractory (R) or relapsed/refractory (RR) acute leukemia (AML n = 66; ALL n = 16) who were treated with clofarabine (10 mg/m2) and cyclophosphamide (200 mg/m2) days 1–4 (ClofCy) prior to HSCT. Results: ClofCy resulted in rapid cytoreduction (median time to granulocytes <0.5 × 109/L: 5 days). HSCT was performed in 62.2% (51/82) of our patients. Infections (18/31, 58%) were the main reason to withhold transplantation. HSCT and non-HSCT patients differed significantly regarding the median number of chemotherapies received prior to ClofCy (HSCT, median = two therapies; non-HSCT, median = three therapies; p = 0.027). Median overall survival (OS) was 0.31 years in all patients (5- and 10-year OS; 24% and 20%, respectively). HSCT patients had a median OS of 1.23 years (5- and 10-year OS; 38.4% and 31%, respectively) and those in the non-HSCT cohort a median OS of 0.11 years (5-year OS; 0%). Median OS and relapse-free survival (RFS) in the HSCT patient cohort were 1.04 and 0.9 years, respectively. Acute graft-versus-host disease (aGvHD) was associated with inferior survival (median OS 0.46 years), whereas chronic graft-versus-host disease (cGvHD) was associated with improved OS and RFS (above 50%). Conclusion: ClofCy is an effective bridging regimen enabling HSCT in patients with R/RR acute leukemia. Introducing ClofCy may improve the probability of reaching HSCT and long-term survival in this cohort of patients with poor prognosis.
SN  - 2072-6694
ER  -