Autologous stem cell transplant (high-dose therapy)
Collect the patient's own blood-forming stem cells, give a very high dose of chemotherapy that would otherwise destroy the marrow, then give the cells back to rebuild it.
High-dose melphalan with autologous stem-cell rescue has been the consolidation standard in transplant-eligible myeloma since the 1990s (IFM 90, Attal 1996) and remains so in the quadruplet era (PERSEUS, IFM 2009/DETERMINATION show PFS but not OS benefit over continued triplet therapy). In lymphoma it is the standard salvage consolidation for chemosensitive late relapse (PARMA) and for relapsed Hodgkin lymphoma; CAR-T has displaced it for early-relapsing LBCL. Tandem (two sequential) transplants are used in high-risk neuroblastoma.
How it works
Peripheral blood stem cells mobilised (G-CSF ± plerixafor), collected by apheresis, cryopreserved; myeloablative conditioning (melphalan 200 mg/m2 in myeloma; BEAM in lymphoma; busulfan-melphalan or carboplatin-etoposide-melphalan in neuroblastoma); reinfusion; engraftment in ~2 weeks.
- Permits dose intensity impossible otherwise
- Decades of outcome data
- Widely available
- Treatment-related mortality ~1-2% (myeloma) to higher in children
- Infertility, second malignancies, prolonged cytopenias
- Being challenged by CAR-T and MRD-guided deferral
Etoposide is a chemotherapy from the mayapple plant, essential to curing testicular cancer (BEP), treating small-cell lung cancer, lymphomas, childhood sarcomas and leukaemias, and used in transplant conditioning.
The myeloma chemotherapy that is also the standard high-dose conditioning before autologous transplant, and, delivered directly into the liver's blood supply or the eye, treats uveal melanoma metastases and retinoblastoma.
PERSEUS, with the earlier GRIFFIN and CASSIOPEIA trials, made a four-drug daratumumab quadruplet the standard for fit patients heading to transplant. It also introduced MRD-directed stopping of the antibody, a step towards treatment that is deep but not indefinite. Whether transplant itself remains necessary on top of a quadruplet is now the open question.
TRANSFORM confirmed ZUMA-7's conclusion with a different CD19 CAR-T and a more permissive design that allowed bridging chemotherapy, making the results closer to real-world practice. Liso-cel's low toxicity makes it attractive for older or frailer patients and for outpatient delivery. Together the two trials made CAR-T the standard second-line therapy for early-relapsing aggressive lymphoma.
ZUMA-7 rewrote second-line treatment for aggressive lymphoma: patients whose disease returns within a year of R-CHOP should be offered CAR-T rather than salvage chemotherapy and transplant. It is also one of the few cell-therapy trials to show an overall survival benefit despite crossover. Patients relapsing later than 12 months were not studied and transplant remains standard for them if chemosensitive.
Latest papers
topQuery for this technology: (TITLE:"Autologous stem cell transplant" OR ABSTRACT:"Autologous stem cell transplant" OR TITLE:"high-dose therapy" OR ABSTRACT:"high-dose therapy") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Autologous stem cell transplant (high-dose therapy), not a curated reading list.
Pages like this
not linked directly; found by shared links- ProductVincristine
Shares Gestational trophoblastic neoplasia, Retinoblastoma, Ewing sarcoma, Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma).
- ProductCyclophosphamide
Shares Gestational trophoblastic neoplasia, Retinoblastoma, Ewing sarcoma, Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma).
- CollectionLymphoma Research Foundation (LRF)
Shares Primary CNS lymphoma, Waldenström macroglobulinaemia, Peripheral T-cell lymphomas (including cutaneous T-cell lymphoma), Mantle cell lymphoma.
- TermAutologous stem cell transplant (ASCT)
Shares Melphalan (including hepatic delivery system), ZUMA-7, Hodgkin lymphoma, Neuroblastoma (paediatric).
- TrialBELINDA
Shares Early relapse: CAR-T before transplant, TRANSFORM: liso-cel CAR-T versus salvage chemotherapy and transplant in early-relapsing large B-cell lymphoma, ZUMA-7: axi-cel CAR-T instead of salvage chemotherapy and transplant for large B-cell lymphoma that relapses early, Diffuse large B-cell lymphoma.
- ProductMethotrexate
Shares Gestational trophoblastic neoplasia, Testicular germ cell tumours, Primary CNS lymphoma, Medulloblastoma.
- ProductBortezomib
Shares Paul G. Richardson, PERSEUS, PERSEUS: daratumumab added to bortezomib-lenalidomide-dexamethasone around autologous transplant in newly diagnosed myeloma, Waldenström macroglobulinaemia.
- TrialTRANSFORM
Shares Early relapse: CAR-T before transplant, TRANSFORM: liso-cel CAR-T versus salvage chemotherapy and transplant in early-relapsing large B-cell lymphoma, ZUMA-7: axi-cel CAR-T instead of salvage chemotherapy and transplant for large B-cell lymphoma that relapses early, Diffuse large B-cell lymphoma.