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HIPS Qualification Testing (HQT): Qualification of Hemophilia Treatment Centers to Perform PBMC Re-Stimulation for the Hemophilia Inhibitor in Previously Untreated Patients (HIPS) Study

1University of Texas Health Science Center at Houston and McGovern Medical School, Houston, United States, 2Texas Southern University, College of Pharmacy and Health Sciences, Houston, United States, 3Baxalta Innovations a Member of the Takeda Group of Companies, Cambridge, United States, 4Baxalta Innovations, a Member of the Takeda Group of Companies, Cambridge, United States, 5Emory University, Children's Healthcare of Atlanta, Aflac Cancer and Blood Disorders Center, Atlanta, United States, 6Maggiore Hospital Policlinico and University of Milan, IRCCS Ca' Granda Foundation, Milan, Italy, 7Rho, Inc., Durham, United States, 8Baxalta Innovations, a Member of the Takeda Group of Companies, Vienna, Austria

Abstract Number: PB0212

Meeting: ISTH 2020 Congress

Theme: Coagulation and Natural Anticoagulants » Coagulation Factors and Inhibitors

Background: HIPS is an international multi-center prospective study to assess immune responses during the first 50 exposure days to FVIII replacement therapy in PUPS with severe hemophilia A. The 15 participating Hemophilia Treatment Centers (HTCs) prepared blood samples for plasma, cellular, epigenetic, genomic, and transcriptome analysis at the local site prior to shipment to central laboratories. FVIII-restimulation of PMBCs was performed at each HTC in order to evaluate antigen-specific cellular immune responses.

Aims: HQT procedures were developed to qualify each clinical site to perform the laboratory procedures prior to subject enrollment in HIPS.

Methods: An on-site demonstration and review was conducted at each qualifying HTC. A test qualification kit consisting of whole blood from two CMV sero-positive donors were sent to each operator for PBMC isolation and restimulation with CMV pp65. A sample from each donor was simultaneously processed at the reference lab for comparison. The test samples were assessed for PBMC yield and viability following isolation procedure. Following restimulation with CMV pp65 IFN-γ gene integrity and concentration was measured. The center operator was considered qualified if the ratio of qualification over reference operator was within 0.67-1.5 for pp65 stimulated – and 0.8-1.25 for positive control -stimulated IFN-γ expression.

Results: Forty operators at 15 HTCs were qualified. 11 of the 15 HTCs had acceptable results on first testing. The other 4 HTCs were below acceptable range, and after a review of procedures were permitted to repeat the testing; all 4 HTCs had acceptable results after the 2nd test.

Conclusions: HTCs are capable of performing quality antigen-specific restimulation testing from PBMCs locally. HQT is a useful methodology to assess quality of HTCs to prepare samples suitable for plasma, cellular, epigenetic, genomic, and transcriptome analysis.


[Sample Table of RNA integrity and Yield ]

To cite this abstract in AMA style:

Brown D, Aboshady I, Donnachie E, Gangadharan B, Hofbauer C, Meeks S, Santagostino E, Bowen J, Reipert B. HIPS Qualification Testing (HQT): Qualification of Hemophilia Treatment Centers to Perform PBMC Re-Stimulation for the Hemophilia Inhibitor in Previously Untreated Patients (HIPS) Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/hips-qualification-testing-hqt-qualification-of-hemophilia-treatment-centers-to-perform-pbmc-re-stimulation-for-the-hemophilia-inhibitor-in-previously-untreated-patients-hips-study/. Accessed September 24, 2023.

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/hips-qualification-testing-hqt-qualification-of-hemophilia-treatment-centers-to-perform-pbmc-re-stimulation-for-the-hemophilia-inhibitor-in-previously-untreated-patients-hips-study/