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. 2021 Oct 10;216(1):43–52. doi: 10.5694/mja2.51284
Medication Funding Patient selection Recommended dose and treatment strategy Response rate Time to response Toxicities
Azathioprine 64
  • General schedule on the PBS

  • PHARMAC‐funded

  • No access to TPO‐RAs or rituximab, and patients who have expressed a preference to avoid surgery

  • Acceptable safety profile in pregnancy 14 , 65

  • 1–3 mg/kg per day (50–200 mg daily)

  • Concomitant steroids are usually required when starting treatment

40–60% 3–4 months
  • Perform TPMT assay to confirm normal enzyme clearance

  • Interaction with allopurinol

  • Nausea, infection and neutropenia

Mycophenolate mofetil 66
  • General schedule on the PBS

  • PHARMAC‐funded

  • No access to TPO‐RAs or rituximab, and patients who have expressed a preference to avoid surgery

  • Start at 250 mg twice a day, double the dose every 2 weeks until response or as tolerated

  • May start higher or increase sooner if less concerned about gastrointestinal side effects

  • Maximum dose 3000 mg per day

50–60% 50% of patients respond by 4 weeks
  • Monitor FBC carefully for cytopenias

  • Diarrhoea is common

  • Other toxicities: neutropenia, anaemia and viral infections

  • Small increased risk of malignancy and progressive multifocal leukoencephalopathy with prolonged use

Hydroxychloroquine 67
  • Streamlined authority; PBS reimbursement as “autoimmune disease”

  • PHARMAC‐funded

  • ANA‐positive and unable to access TPO‐RAs or rituximab

  • Use with caution in patients with pre‐existing heart disease or risk of retinopathy

  • 200 mg twice a day

  • Concomitant steroids are usually required when starting

60% 2–3 months
  • Most common adverse effects include gastrointestinal symptoms and/or rash

  • Rare, but significant adverse effects, include arrhythmias, cardiomyopathy and retinopathy

  • Long term users should have annual ophthalmology review

Danazol 68
  • No reimbursement from PBS

  • In Australia, it needs SAS application to import from international supply

  • No longer generally available in New Zealand

  • Consider in men with no history of prostate cancer, who do not have a history of thromboembolic disease, who have previously responded to corticosteroids, and who are unable to access rituximab or TPO‐RAs

  • 200 mg given two to four times a day. Dose can be tapered once response is obtained

40–50% 3–6 months
  • Increased risk of thrombosis and liver toxicity

  • Androgenic side effects

  • PSA should be checked in men before use

Dapsone 69
  • General schedule on the PBS

  • PHARMAC‐funded

  • No access to rituximab or TPO‐RAs

  • Comorbidities such as thromboembolic disease may make use of TPO‐RAs less desirable

  • Additional benefit as Pneumocystis jiroveci pneumonia prophylaxis for patients who may be at increased risk from immunosuppression

  • 100 mg/day

50% 3 weeks
  • G6PD assay before commencement

  • Monitor haemolysis markers (haptoglobin, reticulocyte count and LDH) to guide effectiveness and toxicity. Ideally, target a small amount of subclinical haemolysis

  • Severe oxidative haemolysis especially in patients with G6PD deficiency

  • Other side effects include abdominal distension, anorexia, and methaemoglobinaemia, which manifests as cyanosis and breathlessness

Ciclosporin 70
  • General schedule on the PBS

  • PHARMAC‐funded

  • No access to TPO‐RAs or rituximab, and patients who have expressed a preference to avoid surgery

  • Acceptable safety profile in pregnancy 71

  • 3–5 mg/kg per day in two divided doses (75–300 mg twice a day)

  • Concomitant steroids are usually required when starting

40–60% 1–3 months
  • Common side effects include hypertension, renal impairment, headaches and infections

  • Trough levels can be monitored but consult with local reference ranges to minimise toxicity

ANA = antinuclear antibodies; FBC = full blood count; G6PD = glucose‐6‐phosphate dehydrogenase; LDH = lactate dehydrogenase; PBS = Pharmaceutical Benefits Scheme (Australia); PHARMAC = Pharmaceutical Management Agency (New Zealand); PSA = prostate‐specific antigen; SAS = Special Access Scheme; TPMT = thiopurine methyltransferase; TPO‐RAs = thrombopoietin receptor agonists.