David McIntosh: Thalassaemia Care Needs More Than Breakthrough Therapies

David McIntosh, Founder and Chair of United Plasma Action, shared The Lancet Haematology’s post on LinkedIn, adding:

”This month’s Lancet editorial (link below) opens with a plea about Thalassaemia treatment that applies to all treatments of all afflictions, everywhere.

It’s a call for thalassaemia progress to be measured by whom it reaches – going on to say that breakthrough therapies exist, but remain out of reach for most patients.

This observation is universal.

Almost everyone agrees that we must ‘put patients first’ – and yet it takes a Lancet editorial to point out to us the simple truth that, shout about it as we may, we’re not actually doing it.

As the editorial goes on to say – closing the gap will take care designed around affordability, self-sufficiency, and collaboration.

Almost everywhere amongst the LMIC nations of our sadly unequal World, all three key factors are either sub-optimally implemented or absent altogether.

For patients, carers and clinicians affected by Thalassaemia – and a thousand other afflictions – in most countries, most of the time, what’s needed most – and most urgently – is not breakthrough technologies.

It’s breakthrough National Willpower!”

The Lancet Haematology shared a post on LinkedIn:

”November Issue Now Live!

This month’s editorial calls for thalassaemia progress to be measured by who it reaches.

Breakthrough therapies now exist, but remain out of reach for most patients. Closing the gap will take care designed around affordability and self-sufficiency, and collaboration that runs both ways.

Our November cover by Vito Ansaldi, is inspired by our new joint Series with The Lancet Regional Health – Southeast Asia, Thalassaemia revisited: from health burden to breakthroughs led by Khaled Musallam.

This Series features the scale of thalassaemia in the WHO South-East Asia region (Frédéric B. Piel); comprehensive prevention programmes across Asia (Michael Angastiniotis); treatment landscape for β-thalassaemia (Khaled Musallam); and new models of care (Hanny Al-Samkari).

A linked comment on the Series by Eugene Fan et al.

A comment on α-thalassaemia by Raffaella Origa et al.

This issue features

Hydroxyurea and infection risk in children with SCD in sub-Saharan Africa

In this retrospective cohort study of 606 children from the REACH trial, non-malarial infection rates fell from screening to the maximum tolerated dose phase (adjusted IRR 0·51), with no increase in infection incidence or severity. Thomas N Williams (Imperial College London) and colleagues.

A linked comment by Nancy Green et al.

Phenotype, treatment, and prognosis in Erdheim–Chester disease

In this retrospective cohort of 1174 patients, BRAF and MEK inhibitors achieved objective response rates of 74% and 71%, yet 22% of patients died, with neurodegeneration (HR 4·2) among the independent predictors of mortality. Julien Haroche (Sorbonne University) & colleagues.

Listen to the authors’ insights.

Decitabine plus busulfan–cyclophosphamide conditioning in KMT2A-rearranged leukaemia

This open-label phase 3 trial of 130 patients found a 2-year cumulative incidence of relapse of 12 percent with decitabine plus busulfan–cyclophosphamide vs 29 percent with busulfan–cyclophosphamide alone (HR 0·41).

Qifa Liu (Southern Medical University) and colleagues.

MK-4002 in relapsed or refractory multiple myeloma

In this first-in-human phase 1 study of 97 heavily pretreated patients, the BCMA-targeting T-cell engager had manageable safety (grade 3 or worse adverse events in 79 percent; cytokine release syndrome in 30 percent with 2 grade 3).

12 mg was identified as RP2D.

Sumit Madan(Banner Anderson Cancer Center) and colleagues.

A linked comment by Holly Lee.

A Review on emerging novel therapies for hereditary haemorrhagic telangiectasia by Hanny Al-Samkari (Massachusetts General Hospital)

In Focus section:

• From survival to self-determination by Kostas Tsourlis
• From pain to purpose by Shaluka Premachandra

Read the Issue.”

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