Canterbury DHB



In This Section

Key Points

Supportive Care

Low Risk MDS

High risk MDS

Allogenic Stem Cell Transplantation

Key Points

Treatment of patients with MDS depends on a number of factors including patient age, comorbidities, and whether they fall into a high risk or low risk group according to their prognostic score. Include the patient in a clinical trial or registry whenever possible.

Treatment options include:

Supportive Care

Low Risk MDS

Erythropoietin in MDS

Erythropoiesis-stimulating agents (ESA) are recommended for the treatment of anaemia in low and intermediate risk MDS (evidence level 1B) by the British Committee for Standards in Haematology, updated in 2014.

From 2014, erythropoietin (EPO) was funded by PHARMAC for patients with:

A validated model for predicting response to EPO has been published:

Validated model for predicting response to erythropoietin (reference)

Transfusion need




< 2 units RBC/month


< 500 units/L


≥ 2 units RBC/month


≥ 500 units/L


RBC = red blood cells

Predictive response to erythropoietin=stimulating agent (ESA):

Treatment is recommended for patients with a score of 0 or 1. A Special Authority is required.

Under the PHARMAC rules, the maximum dose of erythropoietin is 80,000 international units per week and the initial authority application is for 2 months with a renewal if there is evidence of a response.

In New Zealand, from 1 March 2015, the funded form of erythropoietin is Eprex (erythropoietin alpha) and therefore when writing a script you should specify Eprex or erythropoietin alpha (not NeoRecormin or erythropoietin beta). Eprex is only available as 10,000 units/syringe and is given via a subcutaneous injection.

It is recommended that the dose of erythropoietin is rapidly escalated to the maximum dose to ensure a response in the haemoglobin within the initial 2 month period. Haemoglobin levels should not rise above 120 g/L.

Patients with uncontrolled hypertension should not be commenced on ESA and all patients should have regular monitoring, i.e. weekly, of their blood pressure when treatment with ESA is initiated.

Administration of erythropoietin

Patients can be educated to inject themselves. Patient information is available on:

Education can be done by practice nurses in the community or alternatively by the haematology nursing staff on the day ward.

If patients are unable to self-inject, they should be referred to their GP practice nurse or district nursing staff.

Erythropoeitin and GCSF

Patients who have subtypes of MDS with ringed sideroblasts present may respond to the combination of EPO with GCSF. Response rates may be as high as 50%. GCSF is not funded for this indication in New Zealand but patients may wish to pay for this privately. The aim is to double the white cell count or maintain the count between 6-10 x 109/L starting at a dose of 300 micrograms/week in 2 to 3 divided doses.

Iron Chelation

Patients with low risk MDS are at risk of transfusional iron overload. There is an ongoing debate as to the clinical significance of iron loading in this group of patients and given that the majority of patients with MDS are in the older age group at diagnosis it is not a relevant issue for most. For younger patients or those who may be a candidate for stem cell transplantation, consider the potential problems of iron loading.

Although the serum ferritin can be influenced by other factors, most guidelines use this parameter to direct iron chelation therapy. A liver biopsy is the gold standard test for measuring iron loading but this is an invasive test. T2* MRI scanning can be used to assess the degree of cardiac and liver loading but there is not yet a clear correlation between these results and the degree of RBC transfusions or outcome.

Desferrioxamine is the only iron chelator currently available in New Zealand and is given via a subcutaneous injection, either BD or an infusion. Further information can be found on Iron Overload - Transfusion.

Deferasirox (EXJADE™), an oral iron chelator, is also licensed for use in patients with transfusional iron overload. However, it is not currently funded in New Zealand for patients with myelodysplasia. An application can be made via the NPPA system.

Table 2. Recommendations for management of iron overload in MDS patients (page 5) gives a suggested management plan for patients who either have or are at risk of transfusional iron overload (in Shenoy, N., et al. (2014). Impact of iron overload and potential benefit from iron chelation in low-risk myelodysplastic syndrome).


MDS associated with del(5q) is a specific subtype of MDS, which has a relatively indolent course and better prognosis. The median survival is at least 6 years.


List et al Lenalidomide in the Myelodysplastic Syndrome with Chromosome 5q Deletion N Engl J Med 2006;355:1456-65.

Further reading

Killick, S. B., et al. (2014). Guidelines for the diagnosis and management of adult myelodysplastic syndromes. Br J Haematol 164(4): 503-525.

High risk MDS


Azacitidine is a hypomethylating agent and is an alternative to intensive chemotherapy in patients with high risk MDS. The AZA001 study (Fenaux et al, 2009) was the first study to show a significant improvement in overall survival with azacitidine treatment as compared to best supportive care, low-dose cytarabine or intensive AML-type treatment. Median overall survival was 24.5 months vs. 15.0 months (P=0.0001) and 45% of patients became transfusion independent compared to 11% in the conventional treatment group. The median duration of response was 13 months. Data has shown an improved overall survival compared to conventional treatment in patients ≥ 75 years with good performance status.

The majority of patients achieve a response by the end of 6 cycles of treatment, however 48% of all responders continue to improve the level of the response beyond this time point. The optimal number of treatment cycles is not known, however those patients who have responded should continue for as long as the response is maintained. It has been shown that patients who discontinue treatment rapidly relapse with a median time to progression of 5.4 months. Often, these patients do not regain a good response on re-treatment.

Azacitidine is approved by PHARMAC (following a decision in 2014) for the treatment of patients with a performance status (WHO / ECOG) of 0-2, life-expectancy of at least 3 months and:

Patients with secondary myelodysplasia are excluded.

A Special Authority is required.


Azacitidine is given via a subcutaneous injection as an outpatient treatment.

The initial trials used a dosing schedule of 75 mg/m2 for 7 consecutive days (AZA001 Fenaux). However, this is not feasible locally given the logistics of preparing the drug over the weekend. Several regimes have been suggested, with no evidence available that one dosing regime is superior to another. Patients treated in Christchurch receive 7 x 75 mg/m2 doses given daily excluding weekend days.

The following resources are available:

Common toxicities include haematological effects and renal dysfunction. Dose adjustments may be necessary and are documented in the dose adjustment information sheet, which can be found with the chemotherapy chart. For full details, refer to the medicine datasheet.

Injection-site reactions are common and the CDHB has a policy for managing these.

Monitoring patients

Patients should have a CBC every 2 weeks throughout cycle 1 and 2, then at least monthly at the beginning of treatment cycles. Monitor biochemistry, including renal / liver function and bicarbonate, at least monthly at the start of each treatment cycle.

It is recommended that a bone marrow be done at 6 (+12 ) months to assess response, to assess toxicity as detailed in the dose adjustment guidelines, and as clinically appropriate to assess relapse or ongoing response.

Further reading

Fenaux et al. (2009) Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. The Lancet Oncology, 10, 223–232.

Fenaux et al. (2010) Practical use of azacitidine in higher-risk myelodysplastic syndromes: an expert panel opinion. Leukemia Research, 34(11):1410-6.

Seymour et al. (2010) Effects of azacitidine compared with conventional care regimens in elderly (≥ 75 years) patients with higher-risk myelodysplastic syndromes. Critical Reviews in Oncology Hematology, 76, 218–227.

Lyons et al. (2009) Hematologic response to three alternative dosing schedules of azacitidine in patients with myelodysplastic syndromes. Journal of Clinical Oncology, 27, 1850–1856.

Voso et al. (2013) Rapid loss of response after withdrawal of treatment with azacitidine: a case series in patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia. Eur J Haematol, 90, 345–8.

Allogenic Stem Cell Transplantation

The decision to offer intensive therapy is based on the patient’s age, IPSS score, and the availability of a HLA matched sibling donor. A matched, unrelated donor (MUD) stem cell transplant can be considered for the younger patient (under 30-40) with MDS.

For further information, see Deeg, ASH EPB 2005, p. 167 - and Stem Cell Transplantation (SCT).

About this Canterbury DHB document (4960):

Document Owner:

Emma-Jane McDonald (see Who's Who)

Last Reviewed:

November 2019

Next Review:

November 2021


Note: Only the electronic version is controlled. Once printed, this is no longer a controlled document. Disclaimer

Topic Code: 4960