Lay summaries
Lay English summaries of articles published by the EUMDS Registry
The Registry has been opened for a while, and in this timeframe the scientific community has upgraded the criteria which were used to calculate the prognosis of patients with MDS. These new criteria (IPSS-R) were validated on the first 1000 EUMDS patients who were characterized using the ‘’old’’ (IPSS) criteria. This study shows that using the new criteria, our risk assessment improved for the various MDS patient groups. Currently, both the old and the new criteria need to be reported by the physician for each patient who joins the Registry.
The lower-risk MDS population is a highly diverse group of patients. Usually, the prognosis or risk profile is formulated on static variables at time of diagnosis. Therefore, we set out to determine whether there are additional measurements / characteristics during the course of MDS that could be helpful to further tailor the prognosis of a patient in lower-risk MDS patients. We found that a sharp decrease (of more than 25%) of platelets in the first 6 months after diagnosis is a marker for a poorer prognosis. Patients that had such a decrease in the first 6 months after diagnosis were more prone to develop high risk MDS and survive a shorter length of time. Administration of red blood cell transfusions during the first 6 months after diagnosis was an additive and independent indicator of prognosis. A useful aspect of these measurements is that it does not require more blood sampling from the patient besides the regular checkup, and it is inexpensive.
Health-related quality of life (HRQoL) is an important parameter to measure the well-being of a patient or patient group. It can be difficult to reliably capture the impact of a disease on the personal life of a patient, especially in an ageing patient group. We measured HRQoL both in a MDS patient group at diagnosis, and compared this to databases of HRQoL for people from the same age, sex and country (control group). This study shows that patients with MDS have in general a lower HRQoL compared to the control group. Female MDS patients have more problems with anxiety / depression compared to the healthy population. Male MDS patients showed also increase in anxiety / depression issues, but they also more often experienced problems with mobility. Furthermore, older age was associated with an increased impairment in various categories. Overall, MDS patients have a lower quality of life compared to a healthy ‘’matched’’ population.
Red blood cell transfusions (RBCT) are an important part of the general medical care for MDS patients. However, the overall life expectancy of patients treated with RBCT is often decreased compared to untransfused MDS patients. It is unclear whether this lower life expectancy is due to the nature of the disease (more severe illness and therefore greater need for transfusions) or whether it is due to unwanted side effects of the RBCT itself. We observed that even patients transfused with small quantities of blood transfusions have a negatively affected life expectancy. We are unsure what causes this negative association, and hypothesize that it could be related to the release of toxic iron radicals after transfusion. Future studies are needed to confirm these observations and in addition, it is necessary to perform dedicated intervention studies with the aim to overcome this negative effect.
Red blood cell transfusions (RBCT) are an important part of the medical care for MDS patients. A small majority (51%) of the lower-risk MDS patients in the Registry become dependent on these transfusions. Transfusion dependent patients are prone to accumulate iron in the body because of the RBCT, which deposits iron in parts of the body that is out of reach of the usual processes that get rid of excess iron from the body. Those systems can become damaged by overload with iron resulting in negative side effects. In this article we show that transfusion dependent MDS patients have indeed an imbalanced iron control system. This was particularly found in a subgroup of MDS patients with ringed sideroblasts, some of whom had never even become dependent on blood transfusions. Ringed sideroblasts are bone marrow red blood cells which are loaded with iron. In addition, we describe a clinically relevant marker which assists in predicting the survival of these lower-risk MDS patients: labile plasma iron (LPI).
The 17 countries, participating in the Registry, have their own regulations and treatment protocols. Erythropoiesis-stimulating agents (ESA) are not part of the treatment regimen in all countries. ESA is a drug which stimulates the bone marrow stem cells to make red blood cells. This study investigated the differences in ESA use, to examine if this translated to differences in MDS patient outcomes. We observed some differences in the use of ESA across Europe. For example, ESA was not administered at all in some countries. In some countries, it was the general practice to prescribe ESA before the patient had become transfusion dependent. We found that ESA is most effective to treat anemia in lower-risk MDS in patients before they become transfusion dependent. This finding does not support the practice of treating patients with ESA only after they have become transfusion dependent.
Red blood cell transfusions (RBCT) are an important part of the medical care for lower-risk MDS patients. However, these transfusions can cause iron overload in the body. This excess in iron has been associated with other complications for the patient and even increased mortality. Iron chelation is a therapy which removes the excess iron from the body. Both patients with and without iron chelation are present in the Registry. In this study we compared both groups of patients, aiming to assess the effect of iron chelation therapy. We found that patients who have received iron chelation therapy had a superior survival compared to those who did not receive iron chelation. In addition, many patients who were chelated needed less of this transfusions and showed an improvement of anemia. We conclude that iron chelation therapy is effective and can improve the survival in LR-MDS patients