Summary
Busulfan has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Non-Hodgkin Lymphoma (NHL) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Non-Hodgkin Lymphoma (NHL). ClinicalTrials.gov lists 8 registered trials linking Busulfan to Non-Hodgkin Lymphoma (NHL): 1 is currently recruiting; 1 is active or not yet recruiting; 4 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00143845). It plans or enrolled 54 participants. Registration activity spans 1998 to 2020. The literature layer holds 3 publications for this pair: 1 systematic review and 2 randomised controlled trial publications. Publication years run from 2014 to 2021. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Non-Hodgkin Lymphoma ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Indolent NHL (follicular): spontaneous remission in 20-30% of early-stage, allowing watch-and-wait; aggressive NHL (DLBCL): rapidly lethal without treatment in weeks-months.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 54.0 (trials 25.0, literature 9.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 1 active / not yet recruiting, 4 completed, 2 other |
| Linked publications | 3 (2 randomised controlled trial publications, 1 systematic review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | BCR |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT00143845 | Study of Low-Intensity Conditioning for Allogeneic Stem Cell Transplant 2003 | completed | Phase 2 | 54 |
| NCT00238433 | Busulfan, Melphalan, and Thiotepa in Treating Patients Who Are Undergoing an Autologous Stem Cell Transplant for Hodgkin's or Non-Hodgkin's Lymphoma 2005 | completed | Phase 2 | 37 |
| NCT00003661 | Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer 1998 | completed | Phase 2 | 3 |
| NCT01244906 | Post-transplant Cyclophosphamide and Sirolimus Following Reduced Intensity Conditioning (RIC) Transplant 2010 | completed | Phase 2 | 26 |
| NCT04644016 | Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders 2020 | recruiting | Phase 2 | 31 |
| NCT00818961 | Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer 2005 | terminated | Phase 2 | 36 |
| NCT02836639 | BEB Conditioning Regimen for Autologous Stem-cell Transplantation(ASCT) in Non-Hodgkin's Lymphoma 2016 | status unknown | Phase 2 | 20 |
| NCT01961063 | Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma 2015 | active, not recruiting | Phase 1 | 3 |
Published literature
- Systematic review Chemotherapy for non-Hodgkin lymphoma in the hemodialysis patient: A comprehensive reviewYasuda H, Yasuda M, Komatsu N · Cancer science · 2021 · PMID 33938097
- Randomized controlled trial High-dose chemotherapy and autologous stem cell transplant compared with conventional chemotherapy for consolidation in newly diagnosed primary CNS lymphoma--a randomized phase III trial (MATRix)Schorb E, Finke J, Ferreri AJ et al. · BMC cancer · 2016 · PMID 27098429
- Randomized controlled trial Rituximab after lymphoma-directed conditioning and allogeneic stem-cell transplantation for relapsed and refractory aggressive non-Hodgkin lymphoma (DSHNHL R3): an open-label, randomised, phase 2 trialGlass B, Hasenkamp J, Wulf G et al. · The Lancet. Oncology · 2014 · PMID 24827808
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target BCR. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Busulfan approved for Non-Hodgkin Lymphoma (NHL)?
Busulfan has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Non-Hodgkin Lymphoma (NHL) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Non-Hodgkin Lymphoma (NHL). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Busulfan in clinical trials for Non-Hodgkin Lymphoma (NHL)?
ClinicalTrials.gov lists 8 registered trials linking Busulfan to Non-Hodgkin Lymphoma (NHL): 1 is currently recruiting; 1 is active or not yet recruiting; 4 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT00143845). It plans or enrolled 54 participants. Registration activity spans 1998 to 2020.
What does the evidence show for Busulfan in Non-Hodgkin Lymphoma (NHL)?
Busulfan has both completed registered trials and synthesis-level publications linked to Non-Hodgkin Lymphoma (NHL). That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target BCR. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.