TY - JOUR
T1 - A case study of 10 patients administered HBOC-201 in high doses over a prolonged period
T2 - outcomes during severe anemia when transfusion is not an option
AU - Zumberg, Marc
AU - Gorlin, Jed
AU - Griffiths, Elizabeth A.
AU - Schwartz, Garry
AU - Fletcher, Bradley S.
AU - Walsh, Katherine
AU - Dao, Kim Hien
AU - Vansandt, Amanda
AU - Lynn, Mauricio
AU - Shander, Aryeh
N1 - Funding Information:
Dr. Shander received research grants from CSL Behring, Gauss Surgical, Masimo, and HbO2 Therapeutics. He has also received honoraria from CSL Behring, Masimo, and Merck and acted as a consultant for CSL Behring, Gauss Surgical, Masimo Corporation, and Vifor. Dr. Gorlin is on the medical advisory board of Quotient/Mosaic. He is principal investigator for the manufacturing sites for both Praise (pathogen inactivation of red cells) and MiPlate (pathogen inactivation of apheresis platelets).
Funding Information:
information: The Hemopure (HBOC-201) product given to the cases presented here was provided by the manufacturer, HbO2 Therapeutics LLC, at no cost, under the FDA Expanded Access program.
Publisher Copyright:
© 2020 AABB
PY - 2020/5/1
Y1 - 2020/5/1
N2 - BACKGROUND: Hemoglobin-Based Oxygen Carriers (HBOCs) can act as an “oxygen bridge” in acute severe anemia when transfusion is indicated, but not possible. We present data on 10 Expanded Access (EA) patients treated with high cumulative doses of Hemopure (HBOC-201), to assess the ability of HBOC-201 to safely treat life threatening anemia in situations where high volumes of product were administered over an extended period of time. STUDY DESIGN AND METHODS: Inclusion in this study required that the patient receive at least 10 units of HBOC-201 between 2014 and 2017 under the FDA-sanctioned EA program. Depending on a patient's geographical location, treatment with HBOC-201 was obtained through either a single patient emergency Investigational New Drug (IND) application, or an intermediate size population IND. Of the 41 patients who were treated during this period, 10 patients received 10 or more units of the product. Data were obtained from medical records. RESULTS: Treatments with HBOC-201 started within 24 hours of signing consent and were administered at an average rate of 1.99 (SD 0.17) units per day over a mean of 8.2 days (SD 2.9), during which patients received on average 16.2 units (SD 5.7 units) of HBOC-201. The median pre-treatment nadir corpuscular hemoglobin (Hb) concentration was 3.3 (SD 0.9) g/dL and post-treatment Hemoglobin was 7.3 (SD 1.7) g/dL. Common side effects included methemoglobinemia, gastrointestinal symptoms, and hypertension. However, no product-related serious adverse events (SAEs) were noted. All patients survived. CONCLUSIONS: Administration of HBOC-201 over an extended period is a feasible and safe oxygen bridge for severely anemic patients who cannot be transfused with RBC.
AB - BACKGROUND: Hemoglobin-Based Oxygen Carriers (HBOCs) can act as an “oxygen bridge” in acute severe anemia when transfusion is indicated, but not possible. We present data on 10 Expanded Access (EA) patients treated with high cumulative doses of Hemopure (HBOC-201), to assess the ability of HBOC-201 to safely treat life threatening anemia in situations where high volumes of product were administered over an extended period of time. STUDY DESIGN AND METHODS: Inclusion in this study required that the patient receive at least 10 units of HBOC-201 between 2014 and 2017 under the FDA-sanctioned EA program. Depending on a patient's geographical location, treatment with HBOC-201 was obtained through either a single patient emergency Investigational New Drug (IND) application, or an intermediate size population IND. Of the 41 patients who were treated during this period, 10 patients received 10 or more units of the product. Data were obtained from medical records. RESULTS: Treatments with HBOC-201 started within 24 hours of signing consent and were administered at an average rate of 1.99 (SD 0.17) units per day over a mean of 8.2 days (SD 2.9), during which patients received on average 16.2 units (SD 5.7 units) of HBOC-201. The median pre-treatment nadir corpuscular hemoglobin (Hb) concentration was 3.3 (SD 0.9) g/dL and post-treatment Hemoglobin was 7.3 (SD 1.7) g/dL. Common side effects included methemoglobinemia, gastrointestinal symptoms, and hypertension. However, no product-related serious adverse events (SAEs) were noted. All patients survived. CONCLUSIONS: Administration of HBOC-201 over an extended period is a feasible and safe oxygen bridge for severely anemic patients who cannot be transfused with RBC.
UR - http://www.scopus.com/inward/record.url?scp=85084870275&partnerID=8YFLogxK
U2 - 10.1111/trf.15778
DO - 10.1111/trf.15778
M3 - Article
C2 - 32358832
AN - SCOPUS:85084870275
SN - 0041-1132
VL - 60
SP - 932
EP - 939
JO - Transfusion
JF - Transfusion
IS - 5
ER -