Off-label use of recombinant factor viia in patients following bone marrow transplantation

ABSTRACT Recombinant factor VIIa (rFVIIa, NovoSeven) is FDA-approved for the treatment of bleeding in patients with hemophilia A/B with inhibitors. A growing literature suggests that there

may be expanded indications for the use of NovoSeven in patients with significant bleeding who do not have a known factor deficiency. Severe bleeding refractory to standard hematologic or

hemostatic support is common in patients undergoing bone marrow transplantation (BMT). We review our experience with rFVIIa in three patients (8 years 8 months to 19 years, median 13 years)

treated for pulmonary hemorrhage (_n_ = 1), hemorrhagic cystitis (_n_ = 3), and gastrointestinal bleeding (_n_ = 2). Boluses of 90–270 μg/kg rVIIa with subsequent doses of 90 μg/kg every

4–24 h for 3–14 days were given, concurrent with maintaining platelet counts >50 000/mm3. Transient clinical responses in gross hematuria (two patients) and in pulmonary hemorrhage were

noted within several days of starting rFVIIa, but bleeding in a new site in two patients and renewed bleeding of the initial site in the third resulted in discontinuation of the drug. No

toxicity or adverse events were observed while the patients were on rFVIIa treatment. Because of the substantial cost of this product, the lack of adequate monitoring methodology, and the

variability of current dose and dosing intervals, large randomized studies are needed before definitive off-label use in the setting of BMT can be recommended. _Bone Marrow Transplantation_

(2001) 28, 405–407. Access through your institution Buy or subscribe This is a preview of subscription content, access via your institution ACCESS OPTIONS Access through your institution

Subscribe to this journal Receive 12 print issues and online access $259.00 per year only $21.58 per issue Learn more Buy this article * Purchase on SpringerLink * Instant access to full

article PDF Buy now Prices may be subject to local taxes which are calculated during checkout ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional subscriptions * Read our FAQs *

Contact customer support SIMILAR CONTENT BEING VIEWED BY OTHERS RECOMBINANT HUMAN THROMBOPOIETIN DOES NOT PROMOTE PLATELET ENGRAFTMENT IN NEWLY DIAGNOSED MULTIPLE MYELOMA PATIENTS FOLLOWING

AUTOLOGOUS STEM CELL TRANSPLANTATION Article Open access 13 February 2025 BLEEDING OUTCOMES AND FACTOR UTILIZATION AFTER SWITCHING TO AN EXTENDED HALF-LIFE PRODUCT FOR PROPHYLAXIS IN

HAEMOPHILIA A IN AUSTRIA Article Open access 21 June 2021 A BISPECIFIC ANTIBODY APPROACH FOR THE POTENTIAL PROPHYLACTIC TREATMENT OF INHERITED BLEEDING DISORDERS Article Open access 08

February 2024 REFERENCES * Mariani G, Testa MG, Paolantonio T _et al_. Use of recombinant, activated factor VII in the treatment of congenital factor VII deficiencies _Vox Sanguinis_ 1999

77: 131–136 Article  CAS  Google Scholar  * Monroe DM, Hoffman M, Oliver JA, Roberts HR . Platelet activity of high-dose factor VIIa is independent of tissue factor _Br J Haematol_ 1997 99:

542–547 Article  CAS  Google Scholar  * Hedner U . Recombinant activated factor VII as a universal haemostatic agent _Blood Coag Fibrin_ 1998 9: (Suppl. 1) S147–S152 CAS  Google Scholar  *

White B, Martin M, Kelleher S _et al_. Successful use of recombinant FVIIa (Novoseven) in the management of pulmonary haemorrhage secondary to Aspergillus infection in a patient with

leukaemia and acquired FVII deficiency _Br J Haematol_ 1999 106: 254–255 Article  CAS  Google Scholar  * Novo Nordisk Investigator's Brochure. Recombinant Coagulation Factor VIIa 1999

pp 42–47 * Poon M-C, Katsarou O, Huth-Kuehne A _et al_. Recombinant Factor VIIa in congenital platelet bleeding disorders _Proc Am Soc Hematol_ 2000 96: 256a Google Scholar  * Chuansumrit A,

Chantarojanasiri T, Isarangkura P _et al_. Recombinant activated factor VII in children with acute bleeding resulting from liver failure and disseminated intravascular coagulation _Blood

Coag Fibrinol_ 2000 11: (Suppl. 1) S101–S105 Article  CAS  Google Scholar  * Al Douri M, Shafi T, Al Khudairi D _et al_. Effect of theadministration of recombinant activated factor VII

(rFVIIa; NovoSeven) in the management of severe uncontrolled bleeding in patients undergoing heart valve replacement surgery _Blood Coag Fibrinol_ 2000 11: (Suppl. 1) S121–S127 Article  CAS

  Google Scholar  Download references AUTHOR INFORMATION Author notes * JM Wiley Present address: Division of Pediatric Hematology-Oncology, Sinai Medical Center, Baltimore, MD, USA AUTHORS

AND AFFILIATIONS * Division of Pediatric Hematology-Oncology, University of North Carolina School of Medicine, Chapel Hill, NC, USA J Blatt, SH Gold, JM Wiley, PE Monahan & HC Cooper *

Department of Pharmacy, University of North Carolina School of Medicine, Chapel Hill, NC, USA D Harvey Authors * J Blatt View author publications You can also search for this author inPubMed

 Google Scholar * SH Gold View author publications You can also search for this author inPubMed Google Scholar * JM Wiley View author publications You can also search for this author

inPubMed Google Scholar * PE Monahan View author publications You can also search for this author inPubMed Google Scholar * HC Cooper View author publications You can also search for this

author inPubMed Google Scholar * D Harvey View author publications You can also search for this author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS

ARTICLE CITE THIS ARTICLE Blatt, J., Gold, S., Wiley, J. _et al._ Off-label use of recombinant factor VIIa in patients following bone marrow transplantation. _Bone Marrow Transplant_ 28,

405–407 (2001). https://doi.org/10.1038/sj.bmt.1703157 Download citation * Received: 08 February 2001 * Accepted: 25 May 2001 * Published: 18 September 2001 * Issue Date: 01 August 2001 *

DOI: https://doi.org/10.1038/sj.bmt.1703157 SHARE THIS ARTICLE Anyone you share the following link with will be able to read this content: Get shareable link Sorry, a shareable link is not

currently available for this article. Copy to clipboard Provided by the Springer Nature SharedIt content-sharing initiative KEYWORDS * recombinant factor VIIa * bone marrow transplantation *

pulmonary hemorrhage * hemorrhagic cystitis