Risk factors for joint bleeding in severe hemophilia A and B: analysis of the Community Counts longitudinal surveillance cohort

Authors: Manco-Johnson, Marilyn J. ^{1, ∗}, Le, Binh ², Acharya, Suchitra ³, Ahuja, Sanjay ⁴, Chitlur, Meera ⁵, Citla-Sridhar, Divyaswathi ⁶, Fedewa, Stacey Ann ⁷, Isaac, Daniel ⁸, Kulkarni, Roshni ⁸, Schieve, Laura A. ², Sharathkumar, Anjali ⁹, Soucie, J. Michael ^{2, 10}

Source: In Blood Vessels, Thrombosis & Hemostasis October 2024

Clinical and treatment characteristics of infants and toddlers less than 2 years of age with hemophilia

Authors: Han, Jennifer H., Dupervil, Brandi, Mahajerin, Arash, Kulkarni, Roshni, Manco-Johnson, Marilyn, Thornburg, Courtney

Source: In Blood Advances 11 June 2024 8(11):2707-2717

A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial

Authors: Carcao, Manuel, Schiavulli, Michele, Kulkarni, Roshni, Rendo, Pablo, Foster, Meredith, Santagostino, Elena, Casiano, Sandra, Königs, Christoph

Source: In Blood Advances 26 March 2024 8(6):1494-1503

Depression and anxiety in persons with Von Willebrand disease

Authors: Roberts, Jonathan C., Kulkarni, Roshni, Kouides, Peter A., Sidonio, Robert F., Carpenter, Shannon L., Konkle, Barbara A., Wu, Joanne, Ullman, Megan M., Curtis, Randall, Baker, Judith R., Crook, Nicole, Nichol, Michael B.

Source: Haemophilia. 29(2):545-554

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100186842528.0x000001&indx=1&recIds=ETOCvdc_100186842528.0x000001

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science

Authors: Byams, Vanessa R., Baker, Judith R., Bailey, Cindy, Connell, Nathan T., Creary, Melissa S., Curtis, Randall G., Dinno, Alexis, Guelcher, Christine J., Kim, Michelle, Kulkarni, Roshni, Lattimore, Susan, Norris, Keri L., Ramirez, Lucy, Skinner, Mark W., Symington, Susan, Tobase, Patricia, Vázquez, Esmeralda, Warren, Beth B., Wheat, Emily, Buckner, Tyler W.

Source: Expert review of hematology. 16:87-106

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100177533170.0x000001&indx=1&recIds=ETOCvdc_100177533170.0x000001

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research to advance the health of people with inherited bleeding disorders with the potential to menstruate

Authors: Baldwin, Maureen K., Ahmadzia, Homa K., Bartlett, Diane L., Bensen-Kennedy, Debbie, Desai, Vidhi, Haley, Kristina M., Herman-Hilker, Sherry L., Kilgore, Amanda M., Kulkarni, Roshni, Lavin, Michelle, Luckey, Shari, Matteson, Kristen A., Paulyson-Nuñez, Kristin, Philipp, Claire S., Ragosta, Sachiko, Rosen, Kimberly, Rotellini, Dawn, Weyand, Angela C.

Source: Expert review of hematology. 16:71-86

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100177533018.0x000001&indx=1&recIds=ETOCvdc_100177533018.0x000001

Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults

Authors: Konkle, Barbara A., Oldenburg, Johannes, Pasi, John, Kulkarni, Roshni, Nolan, Beatrice, Mahlangu, Johnny, Young, Guy, Brown, Simon A., Pabinger, Ingrid, Shapiro, Amy, Négrier, Claude, Blanchette, Victor, Ragni, Margaret V., Dumont, Jennifer, Lethagen, Stefan

Source: In Research and Practice in Thrombosis and Haemostasis August 2023 7(6)

Recombinant von Willebrand factor and tranexamic acid for heavy menstrual bleeding in patients with mild and moderate von Willebrand disease in the USA (VWDMin): a phase 3, open-label, randomised, crossover trial

Authors: Ragni, Margaret V ^*, Rothenberger, Scott D, Feldman, Robert, Nance, Danielle, Leavitt, Andrew D, Malec, Lynn, Kulkarni, Roshni, Sidonio, Robert, Jr, Kraut, Eric, Lasky, Joseph, Pruthi, Rajiv, Angelini, Dana, Philipp, Claire, Hwang, Nina, Wheeler, Allison P, Seaman, Craig, Machin, Nicoletta, Xavier, Frederico, Meyer, Michael, Bellissimo, Daniel, Humphreys, Gregory, Smith, Kenneth J, Merricks, Elizabeth P, Nichols, Timothy C, Ivanco, Dana, Vehec, Deborah, Koerbel, Glory, Althouse, Andrew D

Source: In The Lancet Haematology August 2023 10(8):e612-e623

Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B

Authors: Shapiro, Amy D., Kulkarni, Roshni, Ragni, Margaret V., Chambost, Hervé, Mahlangu, Johnny, Oldenburg, Johannes, Nolan, Beatrice, Ozelo, Margareth C., Foster, Meredith C., Willemze, Annemieke, Barnowski, Christopher, Jain, Nisha, Winding, Bent, Dumont, Jennifer, Lethagen, Stefan, Barnes, Chris, Pasi, K. John

Source: In Blood Advances 11 July 2023 7(13):3049-3057

First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results

Authors: Königs, Christoph, Ozelo, Margareth C., Dunn, Amy, Kulkarni, Roshni, Nolan, Beatrice, Brown, Simon A., Schiavulli, Michele, Gunawardena, Sriya, Mukhopadhyay, Sutirtha, Jayawardene, Deepthi, Winding, Bent, Carcao, Manuel

Source: Blood. 139(26):3699-3707

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100183245137.0x000001&indx=1&recIds=ETOCvdc_100183245137.0x000001

Quality of life in a large multinational haemophilia B cohort (The B‐Natural study) – Unmet needs remain

Authors: Berntorp, Erik, LeBeau, Petra, Ragni, Margaret V., Borhany, Munira, Abajas, Yasmina L., Tarantino, Michael D., Holstein, Katharina, Croteau, Stacy E., Liesner, Raina, Tarango, Cristina, Carvalho, Manuela, McGuinn, Catherine, Funding, Eva, Kempton, Christine L., Bidlingmaier, Christoph, Cohen, Alice, Oldenburg, Johannes, Kearney, Susan, Knoll, Christine, Kuriakose, Philip, Acharya, Suchitra, Reiss, Ulrike M., Kulkarni, Roshni, Witkop, Michelle, Lethagen, Stefan, Krouse, Rebecca, Shapiro, Amy D., Astermark, Jan

Source: Haemophilia. 28(3):453-461

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100150041713.0x000001&indx=1&recIds=ETOCvdc_100150041713.0x000001

Whole-exome analysis of adolescents with low VWF and heavy menstrual bleeding identifies novel genetic associations

Authors: Sadler, Brooke, Minard, Charles G., Haller, Gabe, Gurnett, Christina A., O'Brien, Sarah H., Wheeler, Allison, Jain, Shilpa, Sharma, Mutka, Zia, Ayesha, Kulkarni, Roshni, Mullins, Eric, Ragni, Margaret V., Sidonio, Robert, Dietrich, Jennifer E., Kouides, Peter A., Di Paola, Jorge ^*, Srivaths, Lakshmi ^*

Source: In Blood Advances 25 January 2022 6(2):420-428

Characteristics, complications, and sites of bleeding among infants and toddlers less than 2 years of age with VWD

Authors: Dupervil, Brandi, Abe, Karon, O'Brien, Sarah H., Oakley, Meredith, Kulkarni, Roshni, Thornburg, Courtney D., Byams, Vanessa R., Soucie, J. Michael

Source: In Blood Advances 27 April 2021 5(8):2079-2086

Natural history study of factor IX deficiency with focus on treatment and complications (B‐Natural)

Authors: Shapiro, Amy D., Ragni, Margaret V., Borhany, Munira, Abajas, Yasmina L., Tarantino, Michael D., Holstein, Katharina, Croteau, Stacy E., Liesner, Riana, Tarango, Cristina, Carvalho, Manuela, McGuinn, Catherine, Funding, Eva, Kempton, Christine L., Bidlingmaier, Christoph, Cohen, Alice, Oldenburg, Johannes, Kearney, Susan, Knoll, Christine, Kuriakose, Philip, Acharya, Suchitra, Reiss, Ulrike M., Kulkarni, Roshni, Witkop, Michelle, Lethagen, Stefan, Donfield, Sharyne, LeBeau, Petra, Berntorp, Erik, Astermark, Jan

Source: Haemophilia. 27(1):49-59

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100130980469.0x000001&indx=1&recIds=ETOCvdc_100130980469.0x000001

A new hemophilia carrier nomenclature to define hemophilia in women and girls: Communication from the SSC of the ISTH

Authors: Galen, Karin P. M., d'Oiron, Roseline, James, Paula, Abdul‐Kadir, Rezan, Kouides, Peter A., Kulkarni, Roshni, Mahlangu, Johnny N., Othman, Maha, Peyvandi, Flora, Rotellini, Dawn, Winikoff, Rochelle, Sidonio, Robert F.

Source: Journal of thrombosis and haemostasis. 19(8):1883-1887

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100125700978.0x000001&indx=1&recIds=ETOCvdc_100125700978.0x000001

Women and girls with haemophilia and bleeding tendencies: Outcomes related to menstruation, pregnancy, surgery and other bleeding episodes from a retrospective chart review

Authors: Chaudhury, Ateefa, Sidonio, Robert, Jain, Nisha, Tsao, Elisa, Tymoszczuk, Justyna, Oviedo Ovando, Mariana, Kulkarni, Roshni

Source: Haemophilia. 27(2):293-304

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100122738114.0x000001&indx=1&recIds=ETOCvdc_100122738114.0x000001

The spectrum and severity of bleeding in adolescents with low von Willebrand factor–associated heavy menstrual bleeding

Authors: Srivaths, Lakshmi ^*, Minard, Charles G., O'Brien, Sarah H., Wheeler, Allison P., Mullins, Eric, Sharma, Mukta, Sidonio, Robert, Jain, Shilpa, Zia, Ayesha, Ragni, Margaret V., Kulkarni, Roshni, Dietrich, Jennifer E., Kouides, Peter A.

Source: In Blood Advances 14 July 2020 4(13):3209-3216

Favorable pharmacokinetics in hemophilia B for nonacog beta pegol versus recombinant factor IX‐Fc fusion protein: A randomized trial

Authors: Escuriola Ettingshausen, Carmen ^*, Hegemann, Inga, Simpson, Mindy L., Cuker, Adam, Kulkarni, Roshni, Pruthi, Rajiv K., Garly, May‐Lill, Meldgaard, Rikke M., Persson, Paula, Klamroth, Robert

Source: In Research and Practice in Thrombosis and Haemostasis April 2019 3(2):268-276

Long‐term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B‐YOND extension study

Authors: Pasi, K. John, Fischer, Kathelijn, Ragni, Margaret, Kulkarni, Roshni, Ozelo, Margareth C., Mahlangu, Johnny, Shapiro, Amy, P'Ng, Stephanie, Chambost, Hervé, Nolan, Beatrice, Bennett, Carolyn, Matsushita, Tadashi, Winding, Bent, Fruebis, Joachim, Yuan, Huixing, Rudin, Dan, Oldenburg, Johannes

Source: Haemophilia. 26(6):e262-e271

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100117554921.0x000001&indx=1&recIds=ETOCvdc_100117554921.0x000001

Higher rates of bleeding and use of treatment products among young boys compared to girls with von Willebrand disease

Authors: Abe, Karon, Dupervil, Brandi, O'Brien, Sarah H., Oakley, Meredith, Kulkarni, Roshni, Gill, Joan Cox, Byams, Vanessa, Soucie, Michael J.

Source: American journal of hematology. 95(1):10-17

Availability: http://explore.bl.uk/primo_library/libweb/action/display.do?tabs=detailsTab&gathStatTab=true&ct=display&fn=search&doc=ETOCvdc_100097888974.0x000001&indx=1&recIds=ETOCvdc_100097888974.0x000001