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Autologous stem cell transplant collects the patient's own blood-forming stem cells, gives melphalan or another chemotherapy at a dose that would otherwise destroy the marrow, then returns the cells to rebuild it. It is standard consolidation in myeloma and for relapsed lymphoma, though CAR-T has displaced it in early-relapsing large B-cell lymphoma.
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.
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.
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Carmustine is a chemotherapy that reaches the brain. It is given by infusion for brain tumours, lymphoma and myeloma, and as a slow-release wafer left in the cavity after a brain tumour is removed.
Defibrotide (Defitelio) is the only approved treatment for hepatic veno-occlusive disease, a life-threatening liver complication of high-dose chemotherapy before stem cell transplant, in patients whose kidneys or lungs are also failing.
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.
Palifermin (Kepivance) is a growth factor injected for three days before and after high-dose chemotherapy and stem cell rescue to reduce the severe mouth ulcers (mucositis) that this treatment causes in people with blood cancers.
Plerixafor (Mozobil) is an injection given with G-CSF to flush stem cells out of the bone marrow into the blood so that enough can be collected for an autologous transplant in myeloma or lymphoma.
Thiotepa is an old chemotherapy with a modern job: at high doses it prepares patients for stem cell transplants, and it is still instilled into the bladder or body cavities for superficial bladder cancer and malignant effusions.
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.
Query 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.
Shares Defibrotide, Study 2005-01, John F. DiPersio, Stem cell clinics and stem cell tourism.
Shares Placental-site trophoblastic tumour and epithelioid trophoblastic tumour, Gestational trophoblastic neoplasia, After a transplant or cell therapy: what to ask for, Germ cell tumours of childhood and adolescence (extracranial and CNS).
Shares Stem cell clinics and stem cell tourism, Iron overload after transplant, Blood counts that do not come back after CAR-T: ICAHT, Rebuilding the immune system after treatment.
Shares Enteropathy-associated T-cell lymphoma, ALK-negative anaplastic large cell lymphoma, Thiotepa, Nodular lymphocyte-predominant Hodgkin lymphoma (nodular lymphocyte-predominant B-cell lymphoma).
Shares Transplant or CAR-T at second line in diffuse large B-cell lymphoma, Extragonadal germ cell tumour, ZUMA-7, Melphalan (including hepatic delivery system).
Shares Nodular lymphocyte-predominant Hodgkin lymphoma (nodular lymphocyte-predominant B-cell lymphoma), Nodal T-follicular helper cell lymphoma, angioimmunoblastic type (angioimmunoblastic T-cell lymphoma), Relapsed and refractory classical Hodgkin lymphoma, Primary CNS lymphoma.
Shares ANBL17P1, ACNS0333, TRIANGLE, Primary large B-cell lymphoma of the testis.
Shares ACNS0333, IELSG32, Enteropathy-associated T-cell lymphoma, MATRix/IELSG43.