Table 1.

Selected oncologic drugs used in the treatment of non-neoplastic diseases^a

Drug	Mechanism of action	Neoplasm (oncology)		Non-neoplasm		Refs
		Dose rangeb and indications	Toxicity profilec	Dose rangeb and indications	Toxicity profilec
MTX		200–2000 mg per dose	Grade 3	7.5–35 mg per week	Grade 0–1	[19,20,22]
	Folate antimetabolite, inhibits DNA synthesis	ALL, breast cancer, head and neck cancer, NHL, lung cancer, osteosarcoma, Trophoblastic neoplasm	Neurological, gastrointestinal and dermatological symptoms. Pulmonary, bone marrow, renal and hepatic toxicity	Crohn's disease, rheumatoid arthritis, JIA, psoriasis, psoriatic arthritis	Gastrointestinal symptoms, transient elevation of liver enzymes, liver dysfunction
Cyclophosphamide	DNA alkylating agent, inhibits DNA synthesis	250–1300 mg per dose	Grade 3	75–250 mg per dose	Grade 0–1	[51]
		ALL, breast cancer, Burkitt lymphoma, HD, NHL, MM, ovarian cancer, retinoblastoma	Gastrointestinal, dermatological and catarrhal symptoms. Bone marrow, renal, hepatic, pulmonary toxicity. Acute haemorrhagic cystitis, infertility	Behcet's syndrome, idiopathic pulmonary fibrosis, ITP, JIA, lupus nephritis, NS, SLE, transplant rejection prophylaxis, multiple sclerosis, Wegener's granulomatosis	Fatigue, gastrointestinal, catarrhal and dermatological symptoms
6-Mercapto-purine	Purine antagonist, inhibitor of DNA and RNA synthesis	150–350 mg per dose	Grade 3	100–150 mg per dose	Grade 1–2	[52]
		ALL, AML, CML	Gastrointestinal and dermatological symptoms. Bone marrow, hepatic and renal toxicity	Crohn's disease, ulcerative colitis	Gastrointestinal symptoms, bone marrow suppression, elevation of liver enzymes
Thalidomided	Mechanism of action is not completely understood. Selectively reduces levels of TNF	200–1200 mg per dose	Grade 2–3	25–100 mg per dose	Grade 0–1	[53]
		Kaposi's sarcoma, MM, malignant glioma, myelodysplastic syndrome, renal cell cancer	Neurological and dermatological symptoms. Bone marrow suppression, increased risk of thrombosis	Behcet's syndrome, Crohn's disease, SLE, DLE	Neurological symptoms
Vincristine	Vinca alkaloid: inhibitor of microtubule formation, stopping cell division	2–3.5 mg per week	Grade 3	2 mg per month	Grade 0–1	[54]
		ALL, HD, malignant glioma, neuroblastoma, NHL rhabdomyosarcoma, Wilms’ tumour	Neurological, gastrointestinal and dermatological symptoms. Bone marrow suppression, neurotoxicity	ITP,TTP	Neurological symptoms
DFMOe	Inhibitor of ornithine decarboxylase	>10 g per dosee	Grade 4	0.4–0.8 g per day for a year	Grade 0–1	[55,56]
Prostate cancer	Diarrhoea, abdominal pain, alopecia and ototoxicity	Chemoprotection against prostate cancer, actinic keratosis
>0.4 g per day for several months	Grade 0–1
As a cream Hirsutism (facial hair)
>5 g per day for 15 days	Grade 1–2
Sleeping sickness (trypanosomiasis)	Gastrointestinal symptoms
Miltefosinef	Inhibitors of phospholipid biosynthesis of cell membrane	x	x	1.5–2.5 mg per kg for 28 days Leishmaniasis	Grade1–2	[57]
Nausea, vomiting, diarrhoea

^aAbbreviations: ALL = acute lymphocytic leukaemia, AML = acute myelogenous leukaemia, CLL = chronic lymphocytic leukaemia, CML = chronic myelogenous leukaemia, DLE = discoid lupus erythematosus, HD = Hodgkin's disease, ITP = idiopathic thrombocytopenic purpura, JIA = juvenile idiopathic arthritis, MM = multiple myeloma, NHL = non-Hodgkin's lymphoma, NS = nephritic syndrome, SLE = systemic lupus erythematosus, TTP = thrombotic thrombocytopenic purpura.

^bAdult doses.

^cGrade 0 = no adverse reaction, Grade 1 = mild adverse reactions, Grade 2 = moderate adverse reactions, Grade 3 = severe adverse reactions, Grade 4 = life-threatening toxicity.

^dNot used in pregnancy because of its effect against foetal growth teratogenicity.

^eDFMO (Difluoro-methyl-ornithine) was used as an experimental drug in cancer but was abandoned because efficacy could only be achieved with doses associated with serious life-threatening toxicity. However, the drug was revived for cancer chemoprotection, hirsutism and trypanosomiasis treatment at low dose.

^fMiltefosine was initially discovered as an antineoplastic drug; however, few studies were carried out in humans to treat cancer, thus detailed information on its toxicity and dose ranges as an antineoplastic is not available.