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Table 12.1 Drugs used in the treatment of leukaemia and lymphoma. 
Mechanism of action Particular side-effects* 
Alkylating agents 
Bendamustine Alylating agent and purine 
analogue 
Myelosuppression 
Cyclophosphamide Cross-link DNA, impede RNA 
formation 
Haemorrhagic cystitis, 
cardiomyopathy, loss of hair 
Chlorambucil Marrow aplasia, hepatic toxicity, 
dermatitis 
Busulfan Marrow aplasia, pulmonary 
fibrosis, hyperpigmentation 
Melphalan Marrow aplasia 
Nitrosoureas (BCNU, CCNU) Renal and pulmonary toxicity 
Cisplatin Intrastrand DNA linkage Renal dysfunction, neurotoxicity, 
ototoxicity 
Antimetabolites 
Hydroxycarbamide (hydroxyurea) Inhibits ribonucleotide 
reductase 
Pigmentation, nail dystrophy, 
skin ulceration 
Methotrexate Inhibit pyrimidine or purine 
synthesis or incorporation 
into DNA 
Mouth ulcers, gut toxicity 
Cytosine arabinoside Inhibits DNA synthesis CNS especially cerebellar 
toxicity and conjunctivitis at 
high doses 
6-Mercaptopurine†, 6-thioguanine† Purine analogue Jaundice, gut toxicity 
Clofarabine Purine analogue Myelosuppression 
Fludarabine 
Inhibit adenosine deaminase 
2-Chlorodeoxyadenosine 
or other purine pathways 
Deoxycoformycin 
Immunosuppression (low CD4 
counts); renal and 
neurotoxicity (at high doses) 
Cytotoxic antibiotics 
Anthracyclines (e.g. 
daunorubicin) 
Hydroxodaunorubicin 
(Adriamycin) 
Mitoxantrone 
Idarubicin 
Bind to DNA and interfere 
with mitosis 
Cardiac toxicity, hair loss 
Bleomycin DNA breaks Pulmonary fibrosis, skin 
pigmentation 
Plant derivatives 
Vincristine (Oncovin®) Spindle damage Neuropathy (peripheral or 
bladder or gut) 
Vinblastine myelosuppression 
Vindesine 
Etoposide Mitotic inhibitor Hair loss, oral ulceration 
Demethylating agents 
Azacytidine, decytabine Inhibit DNA methlytransferase Myelosuppression 
Signal transduction inhibitors 
Imatinib, dasatinib, nilotinib Inhibit tyrosine kinase Myelosuppression, fluid 
retention 
Miscellaneous 
Corticosteroids Lymphoblast lysis Peptic ulcer, obesity, diabetes, 
osteoporosis, psychosis, 
hypertension 
Trans-retinoic acid Induces differentiation Liver dysfunction, skin 
hyperkeratosis, leucocytosis 
and hyperviscosity, pleural or 
pericardial effusion 
Arsenic Induces differentiation or 
apoptosis 
Hyperleucocytosis, cardiac 
α-Interferon Activation of RNAase and 
natural killer activity 
Flu-like symptoms, 
thrombocytopenia, leucopenia, 
weight loss
Mechanism of action Particular side-effects* 
Bortezomid Proteasome inhibition Neuropathy 
L-Asparaginase Deprive cells of asparagine Hypersensitivity, low albumin 
and coagulation factors, 
pancreatitis 
Thalidomide Lenalidomide Immuno-modulation Neuropathy, constipation, 
thrombosis 
Monoclonal antibodies 
Rituximab (anti-CD20) Induction of apoptosis Infusion reactions, 
immunosuppression 
Alemtuzumab (anti-CD52) Lysis of target cell by 
complement fixation 
Infusion reactions, 
immunosuppression 
Lumiliximab (anti-CD23) Induction of apoptosis Infusion reactions, 
immunosuppression 
Ibritumomab (Zevalin®) 
(anti-CD20+90Y) 
Toxicity to bound cell Myelosuppression, nausea 
Mylotarg® (anti-CD33) Kills myeloid cells Myelosuppression 
* Many of the drugs cause nausea, vomiting, mucositis and bone marrow toxicity, and in large doses infertility. Tissue 
necrosis is a problem if the drugs are extravasated during infusion. 
† Allopurinol potentiates the action and side-effects of 6-mercaptopurine.

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Chapter12

  • 1. Table 12.1 Drugs used in the treatment of leukaemia and lymphoma. Mechanism of action Particular side-effects* Alkylating agents Bendamustine Alylating agent and purine analogue Myelosuppression Cyclophosphamide Cross-link DNA, impede RNA formation Haemorrhagic cystitis, cardiomyopathy, loss of hair Chlorambucil Marrow aplasia, hepatic toxicity, dermatitis Busulfan Marrow aplasia, pulmonary fibrosis, hyperpigmentation Melphalan Marrow aplasia Nitrosoureas (BCNU, CCNU) Renal and pulmonary toxicity Cisplatin Intrastrand DNA linkage Renal dysfunction, neurotoxicity, ototoxicity Antimetabolites Hydroxycarbamide (hydroxyurea) Inhibits ribonucleotide reductase Pigmentation, nail dystrophy, skin ulceration Methotrexate Inhibit pyrimidine or purine synthesis or incorporation into DNA Mouth ulcers, gut toxicity Cytosine arabinoside Inhibits DNA synthesis CNS especially cerebellar toxicity and conjunctivitis at high doses 6-Mercaptopurine†, 6-thioguanine† Purine analogue Jaundice, gut toxicity Clofarabine Purine analogue Myelosuppression Fludarabine Inhibit adenosine deaminase 2-Chlorodeoxyadenosine or other purine pathways Deoxycoformycin Immunosuppression (low CD4 counts); renal and neurotoxicity (at high doses) Cytotoxic antibiotics Anthracyclines (e.g. daunorubicin) Hydroxodaunorubicin (Adriamycin) Mitoxantrone Idarubicin Bind to DNA and interfere with mitosis Cardiac toxicity, hair loss Bleomycin DNA breaks Pulmonary fibrosis, skin pigmentation Plant derivatives Vincristine (Oncovin®) Spindle damage Neuropathy (peripheral or bladder or gut) Vinblastine myelosuppression Vindesine Etoposide Mitotic inhibitor Hair loss, oral ulceration Demethylating agents Azacytidine, decytabine Inhibit DNA methlytransferase Myelosuppression Signal transduction inhibitors Imatinib, dasatinib, nilotinib Inhibit tyrosine kinase Myelosuppression, fluid retention Miscellaneous Corticosteroids Lymphoblast lysis Peptic ulcer, obesity, diabetes, osteoporosis, psychosis, hypertension Trans-retinoic acid Induces differentiation Liver dysfunction, skin hyperkeratosis, leucocytosis and hyperviscosity, pleural or pericardial effusion Arsenic Induces differentiation or apoptosis Hyperleucocytosis, cardiac α-Interferon Activation of RNAase and natural killer activity Flu-like symptoms, thrombocytopenia, leucopenia, weight loss
  • 2. Mechanism of action Particular side-effects* Bortezomid Proteasome inhibition Neuropathy L-Asparaginase Deprive cells of asparagine Hypersensitivity, low albumin and coagulation factors, pancreatitis Thalidomide Lenalidomide Immuno-modulation Neuropathy, constipation, thrombosis Monoclonal antibodies Rituximab (anti-CD20) Induction of apoptosis Infusion reactions, immunosuppression Alemtuzumab (anti-CD52) Lysis of target cell by complement fixation Infusion reactions, immunosuppression Lumiliximab (anti-CD23) Induction of apoptosis Infusion reactions, immunosuppression Ibritumomab (Zevalin®) (anti-CD20+90Y) Toxicity to bound cell Myelosuppression, nausea Mylotarg® (anti-CD33) Kills myeloid cells Myelosuppression * Many of the drugs cause nausea, vomiting, mucositis and bone marrow toxicity, and in large doses infertility. Tissue necrosis is a problem if the drugs are extravasated during infusion. † Allopurinol potentiates the action and side-effects of 6-mercaptopurine.