Research

In brief: PBS restrictions for blood cancer therapies; Severe haemophilia A; DOAC dipstick test

Thursday, 4 Feb 2021


Reduced PBS restrictions recommended for blood cancer therapies

PBS prescribing restrictions should be eased for some blood cancer therapies, according to the latest recommendations from the Pharmaceutical Benefit Advisory Committee (PBAC).

At its November 2020 meeting the PBAC recommended decreasing the PBS restriction level for nilotinib and dasatinib to Authority Required (Telephone) for initial treatment of chronic myeloid leukaemia (CML), and to Authority Required (STREAMLINED) for first and subsequent continuing treatment. However it did not recommend an amendment to the PBS restriction level for ponatinib.

For treatment of refractory follicular B-cell non-Hodgkin’s lymphoma the PBAC recommended an amendment to the PBS restriction level for idelalisib to Authority Required (Telephone) for the initial treatment phase, and Authority Required (STREAMLINED) for the continuing treatment phase.

A recommendation to reduce the PBS restriction level for azacitidine was made for patients with acute myeloid leukaemia (AML). However, The PBAC did not recommended an amendment to the PBS restriction level for midostaurin.

Other applications for amendments to PBS restrictions were not recommended by the PBAC. These included:

  • Systemic anaplastic large cell lymphoma (brentuximab vedotin)
  • Multiple myeloma (lenalidomide and pomalidomide)
  • Acute lymphoblastic leukaemia (blinatumomab, dasatinib, inotuzumab ozogamicin, ponatinib).

Severe haemophilia A study investigates bleeding and quality of life

Additional haemostatic options for severe haemophilia A are needed because patients have breakthrough spontaneous and joint bleeding and impaired physical functioning despite high adherence with exogenous Factor VIII (FVIII), researchers say. Findings were presented at the Virtual Congress of the European Association for Haemophilia and Allied Disorders (3‐5 February 2021) from a multinational study involving 294 patients with severe HA (FVIII ≤ 1 IU/dL) receiving prophylactic FVIII who were followed for up to 52 weeks. Quality of Life score were low for physical functioning, treatment concern and consequences of bleeding. Arthralgia and haemophilic arthropathy were reported by 5.8% and 2.0% of participants, respectively. Serious adverse events were reported by 4.8% participants, including haemophilic arthropathy; haematuria; haematoma; and haemorrhoidal, oesophageal, and muscle haemorrhage.

Simple urine dipstick can detect DOACs in emergency settings

An easy-to-use point-of-care test is now being recommended for detecting direct oral anticoagulants (DOACs) in urine, according to a briefing from the UK’s National Institute for Health and Care Excellence (NICE). The DOASENSE DOAC Dipstick is a non-invasive method of detecting DOACs, as opposed to standard laboratory tests that involve collection of a blood sample. The NICE guideline advises using the dipstick in place of those laboratory tests in emergency settings, or before elective surgery for people taking DOACs. Each dipstick test strip contains four test pads to detect direct oral thrombin inhibitors, direct oral factor Xa inhibitors, a validation pad to assess urine colour and help exclude abnormal urine that may affect results, and a validation pad to detect creatinine to exclude impaired renal function. The test offers results in about 10 minutes, which NICE said may lead to more timely medical treatment decisions.

“This could include administering a reversal agent for thrombin or factor Xa inhibitors, or directing a person to surgery,” the brief states.

According to experts who participated in the briefing, the dipstick test is simple to administer and can be done by healthcare assistants or nursing staff. The advice is based on three published studies – though only one multi-centre, prospective trial – including a total of more than 1,000 people taking DOACs.  The prospective study, which was led by the founder of DOASENSE, found the dipstick test was noninferior to liquid chromatography-tandem mass spectrometry in detecting factor Xa inhibitors, and superior to that method in detecting thrombin inhibitors. In commenting for the NICE brief, one expert noted that more patients could have access to testing with the dipstick test, while laboratory-based DOAC testing has limited availability in most hospitals.

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