The value of platelet transfusions and tranexamic acid to prevent bleeding in myelodysplastic syndromes-related thrombocytopenia has been called into question by Australian data.
A retrospective Victorian study comprised 178 adults with MDS, chronic myelomonocytic leukaemia or other MDS/myeloproliferative overlap neoplasms admitted to Monash Health hospitals between 2016 and 2018.
The study, published in the European Journal of Haematology [link here], reported the median platelet count at first presentation was 90 x 109/L.
While 85.5% of patients did not require treatment for thrombocytopenia, 5.6% received outpatient/day admission platelet transfusion alone, 5% received tranexamic acid alone and 3.9% received both platelet transfusion and tranexamic acid .
The median pre-transfusion platelet count was 13 x 109/L. Patients with the lowest platelet counts <20 x 109/L and <10 x 109/L were more likely to receive platelets alone (32% and 30%), TXA (17.9% and 30%) or both (25% and 40%).
“We found a high burden of transfusion in those who received outpatient platelet transfusion with a median transfusion interval of 7 days, and 22% involved two adult platelet doses,” the study said.
About 28% of platelet transfusions were single-donor apheresis platelets, including three patients requiring HLA-matched platelets.
“Over a median follow-up time of 46 weeks, bleeding events requiring or occurring during hospitalisation occurred in 20 (11.2%) patients, with no significant difference between platelet-transfused patients (6/34, 17.7%) and non-transfused patients (14/145, 9.7%, p=.18).”
The most common cause of bleeding was GI bleeding (13 of 25 events). Three patients died of bleeding: one fatal intracranial haemorrhage and two gastrointestinal haemorrhages, with presenting platelet counts of 18, 38 and 153 x 109/L respectively.
Neither presenting platelet count, IPSS-R score, prior tranexamic acid use, prior platelet transfusion nor antiplatelet/anticoagulant use was associated with increased risk of first bleeding event.
No transfusion reactions were reported.
The investigators, led by haematologist Dr Allison Mo of Monash Health, said that further prospective clinical trials were warranted to determine the efficacy of tranexamic acid , prophylactic platelet transfusion and other interventions to prevent bleeding in MDS patients.
“Platelet dysfunction, for example, has been reported in MDS patients and may account for differential bleeding risks in patients, independent of the platelet count or the use of antiplatelet/anticoagulant medications.”
“Further, though IPSS-R score did not predict bleeding risk in this cohort, we did not have data on gene mutation profiling due to the study period being prior to routine use of next-generation sequencing.”
The study said guidelines provide no recommendation on platelet transfusion threshold in MDS patients and there were few studies of platelet thresholds to reduce bleeding in chronic hypoproliferative thrombocytopenia.
There were also no guidelines on tranexamic acid use in hypoproliferative thrombocytopenia and studies in MDS outpatients with chronic thrombocytopenia are lacking.
“In all, these observations indicate that further prospective clinical trials are warranted to determine the efficacy of TXA[tranexamic acid], prophylactic platelet transfusion and other interventions to prevent bleeding in MDS patients.
“Future trials should focus on outcomes important to patients, such as clinically significant bleeding and quality of life, not just laboratory results such as platelet counts.”
“They should also incorporate health economics outcomes, given the costs and other resources required for administering regular platelet transfusions, and the potential burden for patients, including frequent hospital admission.”