Outcome of Recombinant Factor VIIa Use for Alveolar Hemorrhage in Hematopoietic Stem Cell Transplant Recipients.

Background: Hematopoietic Stem Cell Transplantation (HSCT) is being widely used as an adjunct to conventional chemoradiotherapy for many hematologic and solid malignancies and some non-malignant hematologic conditions. Alveolar Hemorrhage (AH), seen in 1 to 19% of HSCT recipients has a mortality of 70–100%. Case reports have suggested that administration of recombinant factor VIIa to patients with AH results in rapid clinical improvement and improved survival. Objectives: To study the impact of recombinant Factor VIIa administration in HSCT recipients with AH at M.D. Anderson Cancer Center.

Methods: A retrospective review of all adult patients who underwent bronchoscopy after HSCT between January 1, 2002 and December 31, 2004 for a presumptive diagnosis of AH. Outcome measures analyzed included requirement for positive pressure ventilation over the six months following bronchoscopy, the length of index hospitalization and survival. Pearson chi-squared tests, Fisher’s exact tests, and Wilcoxon rank sum tests were used for statistical analyses.

Results: Eighty seven patients were diagnosed with AH during the study period. Recombinant factor VIIa was administered to 24 patients with alveolar hemorrhage. All 24 patients received high dose steroids and intensive supportive care. Along with factor VIIa, 16 patients received platelet transfusions to keep platelet count above 50×10⁹/L, 4 received desmopressin (DDAVP), and 3 received aminocaproic acid. Only 63% were alive at 30 days after bronchoscopy and 38% after 6 months. Overall, 17 of the 24 patients (71%) receiving factor VIIa died during the study period. Seventy five percent required positive pressure ventilation within 6 months of diagnosis. The median duration of hospitalization was 11 days (SD ± 10 days). No thrombotic complications were observed in any of these 24 patients. There were no statistically significant differences in median survival, 1 and 6-month survival, requirement for positive pressure ventilation, respiratory failure as the cause of death, or duration of hospital stay after bronchoscopy between patients with AH who received factor VIIa and those who did not (p=>0.6 for all outcomes).

Conclusion: In our study of 24 patients with AH who received recombinant factor VIIa for AH, no significant improvement in survival or alteration in the hospital course was observed. Further prospective studies are needed to clarify the role of Factor VII for this indication.