The effectiveness of platelet transfusions to prevent bleeding in patients with hematologic cancers remains unclear. This trial assessed whether a policy of not giving prophylactic platelet transfusions was as effective and safe as a policy of providing prophylaxis. In patients with hematologic cancers, severe thrombocytopenia frequently develops as a consequence of the disease or its treatment.

1. A No-Prophylaxis Platelet-
Transfusion Strategy
for Hematologic Cancers
Published in NEJM on may 9, 2013
Speaker- Dr. Shadab Ahmad

2. BACKGROUND
• The effectiveness of platelet transfusions to prevent bleeding in
patients with hematologic cancers remains unclear.
• This trial assessed whether a policy of not giving prophylactic platelet
transfusions was as effective and safe as a policy of providing prophylaxis.
• In patients with hematologic cancers, severe thrombocytopenia
frequently develops as a consequence of the disease or its treatment.

3. • Most platelet transfusions are administered as prophylaxis,
• To increase low platelet counts and
• To reduce the risk of bleeding.
• However, the degree to which prophylactic platelet transfusions benefit
patients with severe thrombocytopenia has been unclear.

4. STUDY DESIGN AND OBJECTIVE
• A randomized, parallel-group, openlabel, noninferiority trial at 14
hematology centers in the United Kingdom and Australia were
conducted.
• The primary objective was to determine whether a policy of not giving
platelet transfusions as prophylaxis against clinical bleeding was as safe
and effective as the provision of prophylaxis.

5. ELIGIBILITY CRITERIA
• People>=16 years of age
• On chemotherapy or stem-cell transplantation for hematologic
cancers
• Expected to have thrombocytopenia (platelet count, <50×10^9 per
litre) for at least 5 days.

6. EXCLUSION CRITERIA
• A previous bleeding episode of WHO grade 3 or 4,
• A bleeding episode of WHO grade 2 during the current admission,
• An inherited haemostatic or thrombotic disorder,
• A requirement for therapeutic doses of anticoagulant agents,
• A diagnosis of acute promyelocytic leukemia,
• Known HLA antibodies, or
• Prior randomization in this trial.

7. INTERVENTION
• Patients were randomly assigned to receive either prophylactic platelet
transfusions, or no prophylaxis, if the platelet count was less than
10×10^9per litre.
• In both trial groups, platelet transfusions were given therapeutically for
bleeding, given before invasive procedures, or given at the clinician’s
discretion.

8. RANDOMIZATION
Patients were randomly assigned to the two study groups in a 1:1 ratio.

9. DATA COLLECTION
• Data collection continued for 30 days after randomization.
• Patients who were discharged home during the follow-up period
completed bleeding diaries.
• If patients reported bleeding, clinical bleeding-assessment forms were
completed at the next hospital visit.
• To ensure the accuracy and uniformity of bleeding assessments, all written
descriptions of bleeding episodes were examined by two assessors who were
unaware of the treatment assignments.

10. STUDY POPULATION

11. PRIMARY END POINTS
Percent patients having bleeding events of WHO grade 2,3 or 4 up to
30 days after randomization

12. SECONDARY END POINTS
1. Bleeding:-
• The number of days with bleeding episodes of WHO grade 2, 3, or 4 during
follow-up was higher in the no-prophylaxis group than in the prophylaxis
group.
• The time to the first bleeding episode was significantly shorter in the no-
prophylaxis group than in the prophylaxis group.
2. Platelet Transfusions:-
• The proportion of patients who received platelet transfusions was lower in
the no-prophylaxis group than in the prophylaxis group.

13. 3. Other Outcomes:-
• A small but significant increase in the number of red-cell units transfused per
patient was seen in the no-prophylaxis group.
4. Subgroup Analysis:-
• A predefined subgroup analysis showed a significant interaction between
patients who underwent autologous stem-cell transplantation and other
patients (P = 0.04).
5. Serious Adverse Events:-
• The proportion of patients with serious adverse events (including sepsis and
respiratory deterioration) did not differ significantly between the study
groups.

15. RESULTS
• Bleeding events of WHO grade 2, 3, or 4 :
50% of patients in no-prophylaxis group (151 of 300 patients)
43% in prophylaxis group (128 of 298 patients).
• 90% confidence interval.
• P = 0.06 for non-inferiority.
Therefore, this study did not show that a no-prophylaxis strategy for
platelet transfusions was non-inferior to a prophylaxis strategy in
relation to the frequency of bleeding events of WHO grade 2, 3, or 4.

16. CONCLUSIONS
• The results of this study support the
need for the continued use of
prophylaxis with platelet transfusion
and show the benefit of such
prophylaxis for reducing bleeding ,as
compared with no prophylaxis.
• However, a significant number of
patients had bleeding despite of prophylaxis.

17. RELEVANCE
• This study suggests the usefulness of prophylactic platelet transfusion
to avert major bleeding in conditions where platelet transfusion is
otherwise not known to worsen thrombocytopenia.
• But, more robust studies are required for various causes of
thrombocytopenia across different population groups before its
usefulness is established.

18. THANK YOU