News in brief: PBS listings for PNH treatments; Australian tool improves subtyping of B-cell ALL; New diagnostic algorithm for DVT reduces need for ultrasound by half

Thursday, 24 Feb 2022


PBS listings for PNH treatments

PBS listing changes for eculizumab (Soliris) and ravulizumab (Ultomiris) for the treatment of paroxysmal nocturnal haemoglobinuria will come into effect from 1 March 2022.

While previously available only under the Life Saving Drugs Program (LSDP) or via clinical trials, eculizumab will be available as PBS Section 100 HSD-subsidised treatments for some eligible patient groups. Access to eculizumab via the LSDP will end, with a three month transition period to PBS access for existing patients  in conjunction with the sponsor company, Alexion.

A new listing for ravulizumab will apply from 1 March for PNH.

The Department of Health says eligibility criteria for PBS-subsidised access to PNH treatments will largely align with the criteria for LSDP-funded access. However the  department emphasises that it is important for clinicians to select the correct item code when seeking authority to prescribe either eculizumab or ravulizumab.

The drugs are reported to be some of the most expensive on the market, with treatment reported to cost around $500,000 per patient, per year.


New tool for subtyping B-cell ALL

Researchers at the South Australian Health and Medical Research Institute (South Australian Health and Medical Research Institute, Adelaide, have developed a tool that predicts acute lymphoblastic leukemia (ALL) subtypes and driver genes from unadjusted mRNA-seq read counts and which can be integrated into current transcriptomic analyses.

Developed in a set of 1,279 B-cell ALL patients in the US and then validated in a cohort of  767 Australian patients, the Allspice tool was found to robustly identify six ALL subtypes that covered one third (32%) of patients by mRNA-seq (PPV ≥ 87%).

“These associations are unambiguous and provide diagnostic information that is often quicker and easier to obtain compared to fusion callers or cytogenetic tests,” they say in a paper published in medRxiv.

For another 40% of patients, five other frequent subtypes were distinguishable, although overlapping transcriptional profiles led to lower accuracy (52% ≤ PPV ≤ 73%).

“Allspice can help oncologists to determine the most likely causal drivers with greater confidence and identify potential therapeutic targets in a shorter time frame,” they concluded


New diagnostic algorithm for DVT reduces need for ultrasound by half

Adjustment in interpretation of D-dimer results can reduce the need for ultrasound imaging to exclude DVT in the lower extremities, according to a Canadian study.

A novel algorithm for the diagnosis of DVT was developed with higher D-dimer cut-offs to determine the need for repeat proximal ultrasound imaging.

It was evaluated in 1508 patients investigated for suspected proximal DVT, of whom 173 (11.5%) had DVT on scheduled diagnostic testing.  The results suggested that DVT can be excluded by D-dimer <1000 ng/mL in patients with low clinical pretest probability and D-dimer <500 ng/mL in those with moderate probability.

Compared with a traditional DVT testing strategy, the new diagnostic approach reduced the need for ultrasonography by 47% from a mean of 1.36 to 0.72 scans/patient, the researchers reported in a paper published in the BMJ.

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