Archive for category: Medication

You are here: / Medication

Myelofibrosis with cytopenias – recommendations for routine clinical practice

/in , , /by

A letter in Leukemia has just been published with recommendations from a global consensus group about management of  myelofibrosis (MF) patients with cytopenias. The authors explain that:

‘National and international guidelines exist for the management of MF; however, a need remains for practical guidance applicable in everyday clinical practice, especially for patients experiencing cytopenias or potential failure of current therapy. The landscape is further complicated by the availability of multiple prognostic tools for MF; as such, clinicians may find disease prognostication challenging and confusing. Additionally, to maximize clinical applicability of trial data, inclusivity of eligibility criteria in the context of the real-world MF patient population should be considered.’

‘Recognizing these significant challenges, an international expert consensus group was established to provide best practice recommendations for healthcare professionals, intending to supplement, but not replace, existing guidelines.

25 key clinical questions across five consensus themes were identified:

  1. Defining the thresholds for anemia, and when to initiate/modify treatment
  2. Defining the threshold for thrombocytopenia and when to initiate/modify treatment
  3. Defining JAKi failure and what would warrant switching treatment
  4. How and when to determine prognosis in patients with MF
  5. Unmet needs in MF clinical trials

Consensus recommendations.

Consensus guidelines – see Table 1
Current and emerging treatments for anemia in myelofibrosis – see Table 2

‘In this collaboration between an international panel of physicians with expertise in MF and a diverse Extended Faculty of hematologists and patients, a high level of consensus was achieved on recommendations addressing a wide range of critical gaps in MF management. These recommendations provide a valuable framework to support clinicians in optimizing care for patients with MF’

A link to the full letter is available HERE.

 

Share to:

Updated British guidelines on myelofibrosis

/in , /by

The British Society for Haematology has just released (November and December 2023) new guidelines for myelofibrosis.

As these are British guidelines, not all treatment options are available in Australia and Australian practices may vary.
However the guidelines provide useful insight into diagnosis and prognosis considerations as well as the growing array of treatment options becoming available for myelofibrosis patients.

There are two parts to the guidelines:

The management guideline includes detailed advice on specific situations such as:

  • Management of myelofibrosis-associated anaemia
  • Management of thrombocytopenic patients
  • Role of splenectomy in myelofibrosis
  • Myelofibrosis in paediatric, teenage and young adult population
  • Myelofibrosis and pregnancy
  • Allogeneic stem cell transplantation for myelofibrosis
 

 

 

Share to:

Myelofibrosis drug Momelotinib approved by the US FDA

/in , , , /by

Here is an Australian news item about the US FDA approval of Momelotinib, a new drug for Myelofibrosis. This is significant as there are few treatment options for those with myelofibrosis, and this is another option for those for whom other therapies are unsuitable. We would love to see it made available to Australian patients in the future.

 

 

Share to:

Interferons in MPNs: a conversation with an MPN specialist

/in , , /by

Anne Brazeau, the founder and CEO of MPN Education and Advocacy International has kindly made available an excellent wide ranging video about interferon use in MPNs.

  • The video showcases a presentation about interferons by Gabby Hobbs, MPN specialist and Clinical Director of Leukemia Service at Massachusetts General Hospital as well as being an Assistant Professor at Harvard Medical School.
  • Dr Hobbs discusses issues that haematologists take into account when considering interferon use in pregnancy. (35 minutes into the video).
  • Dr Hobbs also responds to a range of questions from MPN patients about interferon.

Note that while the video refers often to Besremi which is a longer acting pegylated interferon available in the USA, Europe, and some other countries, Pegasys is also a long acting interferon and Australians are extremely fortunate that it is available on Australia’s Pharmaceutical Benefits Scheme (PBS).

Share to:

New class of drugs being developed for treatment of polycythaemia vera

/in , /by

An update on the progress with these newly developed drugs is provided by Associate Professor David Ross, Consultant Haematologist at Royal Adelaide Hospital and Flinders Medical Centre, Adelaide
————————————————————————————
Treatment options for polycythaemia vera (PV) are limited at present. Hydroxyurea, a mild chemotherapy tablet, is the most widely used drug treatment in Australia, although the use of interferon is increasing since the recent PBS listing of Pegasys®. Ruxolitinib has shown good results in clinical trials in PV, but in Australia is funded only for the treatment of myelofibrosis.

In 2012 a New York research group reported that the combination of a new drug, ‘nutlin’, with interferon suppressed the growth of PV cells collected from patients.1 They did not treat patients with nutlin, but a related compound being developed by Roche has entered clinical trials as idasanutlin.

Idasanutlin is an inhibitor of MDM2, a protein involved in regulating cell growth and survival. Inhibition of MDM2 has a greater effect on the growth of PV cells than of healthy blood cells. A small Phase 1 clinical trial involving 11 PV patients treated with two different oral doses of idasanutlin was recently published in Blood, one of the leading haematology journals.2 The authors reported improved blood counts, reduction in spleen size, improved symptoms, and a reduction in the level of the JAK2 mutation in the blood.  Preliminary results from the phase 1 trial indicated that the main side effects were diarrhoea and nausea, which were usually mild and occurred in around half of people taking the drug.

A Phase 2 clinical trial of idasanutlin is now recruiting PV patients in whom hydroxyurea has caused intolerable side effects or did not achieve satisfactory disease control.3  This study is open at two Australian sites: the Royal Adelaide Hospital in South Australia and the Peter MacCallum Cancer Centre in Victoria.

Other MDM2 inhibitors are in development, including KRT232 from Kartos Therapeutics. KRT232 is being tested in PV and myelofibrosis internationally, and may become available at some Australian clinical trial sites in the coming months.

References:
1. Lu M et al. Combination treatment in vitro with Nutlin, a small-molecule antagonist of MDM2, and pegylated interferon-2a specifically targets JAK2V617F-positive polycythemia vera cells Blood. 2012;120:3098-3105.
2. Mascarenhas J et al. Oral idasanutlin in patients with polycythemia vera Blood 2019; online June 5 doi: 10.1182/blood.2018893545.
3. Available at: https://www.australianclinicaltrials.gov.au

If you would like to know about the trial, please discuss this trial with your GP or specialist. You can also search for details on the Australian Clinical Trials website by typing idasanutlin into the ‘search for a clinical trial’ category.

Share to:

Ropegintererfon Alfa 2B Approved for Marketing for Treatment of Polycythemia Vera in the EU

/in , , /by

Ropeginterferon alfa 2b is a longer acting form of pegylated interferon. It has very recently been approved for marketing in the EU for the treatment of Polycythemia Vera under the name “Besremi”. Read the full news announcement here.

Share to:

Patient Power interview with Nathalie Cook and Ken Young

/in , , /by

Andrew and Esther Schorr from Patient Power in the USA have long been strong advocates for patient knowledge and empowerment. They recently visited Australia and New Zealand as part of their advocacy work.

As Andrew has an MPN (myelofibrosis), he very much understands the issues and challenges involved in living with an MPN.  He and Esther met with many Australian and New Zealand MPN patients and some haematologists. Andrew and Esther have prepared a video about two of those patients’ experiences with advocacy,  MPN AA’s Nathalie Cook and Ken Young.  The video discusses in particular the interferon story and all the effort involved in listing Pegasys, a pegylated-interferon, which is a longer longer-acting formulation of Interferon alpha, onto the Australian Pharmaceutical Benefits Scheme.

The video of their interview is available HERE.

 

Share to:
Translate »