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Finding the right numbers for myelodysplastic syndromes

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Inga Mandac Rogulj, MD

5 March 2019

“Cure sometimes, treat often, comfort always”

Few years ago, I read interesting interview with dr James Watson. He said "We should cure cancer...we should have the courage to say that we can really do it".

One of the most important priorities worldwide today is war against cancer. Today, many countries are increasing their efforts to ensure that all cancer patients receive the best possible care. As Ferlay and colleagues recently published, there were an estimated 3.9 million new cases of cancer and 1.9 million deaths from cancer in Europe in 2018. The most common cancer sites were cancers of the female breast, followed by colorectal, lung and prostate cancer, with the most common causes of death from cancer were cancers of the lung, colorectal, breast and pancreatic cancer.

Population-based cancer registries provide data by collecting individual information on all patients diagnosed with cancer in the general population and give additional clinical variables to provide  data on cancer treatment and survival. Decades ago, the role of cancer registries was limited to describe the burden of the disease, trends, and geographical comparisons. In the meantime, most of the cancer registries extended their data collection to provide survival data to assess the overall efficacy of the health care system. At present time, registries provide great burden of information for the evaluation of generalisation of good practices, the disparities in care and long-term effects of treatment. Critical public health decision-making is dependent upon registry data.

In June 2018, Prof. Savic reported that the situation in Eastern Europe regarding MDS management, with the exclusion of a majority of EU countries, is not very favourable. There is a low level of reimbursement, as well as lower transplant activity.

I believe that national MDS registries provide insights into more precise numbers of those diagnosed with MDS, what the incidence of various types of MDS are, and how those patients with MDS are being treated. In addition, registries inform us about environmental and socioeconomic risk factors, and give insights in possible etiology of the disease according to specific geographical region. Registry could serve as an important tool through the process of negotiations with health insurance fundings/societies to provide more reimbursed drugs for MDS patients.

The Croatian cancer registry has been founded in 1959 with the aim to investigate specific geographical distribution of cancer incidence by site – esophageal cancer, gastric cancer, colonic cancer, rectal cancer, biliary bladder cancer, lung cancer, cancer of the mammae, melanoma, cancer of the prostate, urinary bladder cancer, renal cancer, thyroid cancer, and acute myeloic leukemia.The Croatian Institute of Public Health provided the data that for the year 2018 with the forecast of international cancer research agencies of 25,221 new cases of cancer in Croatia, and 14,439 deaths caused by malignant diseases. The Myelodysplastic syndrome working group of the Croatian Cooperative Group for Hematologic Diseases (CROHEM) was established in November 2012 and published the first Croatian guidelines for diagnosis and treatment of myelodysplastic syndrome (MDS) in 2017. In February 2015, the MDS working group has created registry for MDS patients as part of CROHEM with the data arranged through a questionnaire that MDS working group devised. The questionnaire includes demographic data, patient characteristics (performance status, comorbidities, significant previously received treatment) at diagnosis, disease characteristics at diagnosis, treatment and follow up.

Treatment of anaemia is the major requirement in patients with low-risk MDS and, as expected, in our registry a majority of patients has been treated with RBC transfusions. In patients with low risk MDS and moderate to severe anaemia, low erythropoietin levels and low RBC-transfusion-requirement, treatment with ESAs, with or without granulocyte colony-stimulating factors should be considered. As seen in previous comments, experience with ESA treatment in MDS patients varies between European countries. Efficacy of hypomethylating agents, particularly azacitidine, has changed the treatment paradigm for patients with high-risk MDS. "Real world" evaluation of 5-azacitidine remains unknown, but the recent published evaluation of 1101 high-risk MDS patients has shown that the OS is higher if the  patients receive at least 4 cycles of treatment, indicating the necessity of sustained administration until therapeutic benefits are realised.

But, are these data enough to provide better treatment and to impact decisions of health care insurance companies?

We know that MDS is a relatively rare disease and to have answers to all relevant questions, more real-world data (RWD) on all subtypes of MDS should be collected and analysed with the help of patient registries.

The way the registries are used for different purposes is a question for the clinicians, the researchers and the patients, and how to involve patients and families in patient centred care.

MDS patient support groups are important to raise awareness of MDS, provide  information to patients and families in order to increase the quality of life and make treatments available to those affected by MDS.  

In conclusion, population-based registries have underestimated the incidence of MDS, so there is a high importance of ‘real-world’ analyses of MDS incidence, worldwide treatment strategies and outcomes, where MDS registries are unmet need.

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