Castaman, G., Bona, E.D., Schiavotto, C., Trentin, L., D'Emilio, A., & Rodeghiero, F. (1997). Pilot study on the safety and efficacy of desmopressin for the treatment or prevention of bleeding in patients with hematologic malignancies. Haematologica, 82, 584–587.

To determine if desmopressin use is effective to prevent bleeding episodes for patients with thrombocytopenia due to bone marrow failure or idiopathic thrombocytopenic purpura (ITP)

Desmopressin was delivered as 0.4 microgram/kg diluted in 100 mL of NS infused over 30 minutes. Bleeding time was assessed (using standard procedure) before the start of infusion and at one and four hours postinfusion. It is not clear how patients were chosen for inclusion in this study but all had a bleeding event.

• N = 15
• MEDIAN AGE = 55.5 years (range = 24–82 years)
• MALES: 80%, FEMALES: 20%
• KEY DISEASE CHARACTERISTICS: Acute leukemia, blast crisis of CML, refractory anemia, non-Hodgkin’s lymphoma
• OTHER KEY SAMPLE CHARACTERISTICS: Majority of patients were receiving chemotherapy or just following therapy. Patients had a bleeding episode or were going for a procedure where the risk of bleeding was high.

• SITE: Single-site  
• SETTING TYPE: Outpatient  
• LOCATION: Italy

• PHASE OF CARE: Transition phase after active treatment
• APPLICATIONS: Elder care 

• Pilot study

• Bleeding time was calculated using the Simplate II device.

All of the patients had a favorable response after one infusion of desmopressin. Bleeding time slowed during infusion and stopped usually within one hour. No nasal packing was required. Patients were able to have scheduled dental procedures, none required platelet transfusions, and no significant side effects were noted from the desmopressin. None of the patients had evidence of disseminated intravascular coagulation.

The findings of this pilot study are interesting to consider for hematologic malignancy groups of patients who receive multiple platelet transfusions and are at risk to become refractory to platelets. Further experimentation is needed to validate the results found here, but on a case-by-case basis, desmopressin infusion could be helpful to decrease bleeding time in thrombocytopenic patients.

• Small sample (< 30)
• Baseline sample/group differences of import
• Risk of bias (no control group)
• Risk of bias (no blinding) 
• Risk of bias (no random assignment) 
• Risk of bias (no appropriate attentional control condition) 
• Risk of bias (sample characteristics)
• Measurement validity/reliability questionable
• Findings not generalizable

At this time, this pilot study does not warrant a change in practice due to its severe limitations of generalizability. There needs to be randomized, clinical trials before a change in practice can be endorsed.