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1. Graft-versus-host disease after anti-CD19 chimeric antigen receptor T-cell therapy following allogeneic hematopoietic cell transplantation: a transplant complications and paediatric diseases working parties joint EBMT study: IMMUNOTHERAPY

Author

Ortí, Guillermo, Peczynski, Christophe, Boreland, William, O’Reilly, Maeve, von Bonin, Malte, Balduzzi, Adriana, Besley, Caroline, Kalwak, Krzysztof, Ryhänen, Samppa, Güngör, Tayfun, Wynn, Robert F., Bader, Peter, Mielke, Stephan, Blaise, Didier, Amrolia, Persis, Yakoub-Agha, Ibrahim, Calkoen, Friso, Schubert, Maria-Luisa, Potter, Victoria, Pichler, Herbert, Kröger, Nicolaus, Kwon, Mi, Sengeloev, Henrik, Torrent, Anna, Chalandon, Yves, van Gorkom, Gwendolyn, Koenecke, Christian, Graham, Charlotte, Schoemans, Helene, Moiseev, Ivan, Penack, Olaf, and Peric, Zinaida

Published
2025
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2. Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP

Author

Wachowiak, Jacek, Galimard, Jacques-Emmanuel, Dalissier, Arnaud, Rihani, Rawad, AlSaedi, Hawazen, Wynn, Robert F., Dalle, Jean-Hugues, Peffault de Latour, Régis, Sedlacek, Petr, Balduzzi, Adriana, Schroeder, Thomas, Bodova, Ivana, Gonzalez Vicent, Marta, Gruhn, Bernd, Hamladji, Rose-Marie, Krivan, Gergely, Patrick, Katharine, Sobkowiak-Sobierajska, Agnieszka, Stepensky, Polina, Unal, Ali, Amrolia, Persis, Perez Martinez, Antonio, Rialland, Fanny, Aljurf, Mahmoud, Isgro, Antonella, Toren, Amos, Bierings, Marc, Corbacioglu, Selim, and Kałwak, Krzysztof

Published
2024
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3. Intention-to-treat outcomes utilising a stringent event definition in children and young people treated with tisagenlecleucel for r/r ALL through a national access scheme

Author

Oporto Espuelas, Macarena, Burridge, Saskia, Kirkwood, Amy A., Bonney, Denise, Watts, Kelly, Shenton, Geoff, Jalowiec, Katarzyna A., O’Reilly, Maeve A., Roddie, Claire, Castleton, Anna, Clesham, Katherine, Nicholson, Emma, Alajangi, Rajesh, Prabhu, Shilpa, George, Lindsay, Uttenthal, Ben, Gabelli, Maria, Neill, Lorna, Besley, Caroline, Chaganti, Sridhar, Wynn, Robert F., Bartram, Jack, Chiesa, Robert, Lucchini, Giovanna, Pavasovic, Vesna, Rao, Anupama, Rao, Kanchan, Silva, Juliana, Samarasinghe, Sujith, Vora, Ajay, Clark, Peter, Cummins, Michelle, Marks, David I., Amrolia, Persis, Hough, Rachael, and Ghorashian, Sara

Published
2024
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6. Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCT

Author

Schuetz, C., Gerke, J., Ege, M., Walter, J., Kusters, M., Worth, A., Kanakry, J. A., Dimitrova, D., Wolska-Kuśnierz, B., Chen, K., Unal, E., Karakukcu, M., Pashchenko, O., Leiding, J., Kawai, T., Amrolia, P. J., Berghuis, D., Buechner, J., Buchbinder, D., Cowan, M. J., Gennery, A. R., Güngör, T., Heimall, J., Miano, M., Meyts, I., Morris, E. C., Rivière, J., Sharapova, S. O., Shaw, P. J., Slatter, M., Honig, M., Veys, P., Fischer, A., Cavazzana, M., Moshous, D., Schulz, A., Albert, M. H., Puck, J. M., Lankester, A. C., Notarangelo, L. D., and Neven, B.

Published
2023
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7. Incremental donor lymphocyte infusion to treat mixed chimerism after allogeneic stem cell transplantation in children with non-malignant diseases

Author

Gabelli, Maria, Stepensky, Polina, Ottaviano, Giorgio, Mullanfiroze, Khushnuma, Lazareva, Arina, Zaidman, Irina, Even-Or, Ehud, Lucchini, Giovanna, Chiesa, Robert, Silva, Juliana, Adams, Stuart, Kricke, Susanne, Finch, Maria, Hill, Annette, Mead, Rachel, Veys, Delphine, Schejter, Yael Dinur, Eddin, Adeeb Naser, Worth, Austen, Amrolia, Persis J., and Rao, Kanchan

Published
2023
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8. Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP

Author

Wachowiak, J, Galimard, J, Dalissier, A, Rihani, R, Alsaedi, H, Wynn, R, Dalle, J, Peffault de Latour, R, Sedlacek, P, Balduzzi, A, Schroeder, T, Bodova, I, Gonzalez Vicent, M, Gruhn, B, Hamladji, R, Krivan, G, Patrick, K, Sobkowiak-Sobierajska, A, Stepensky, P, Unal, A, Amrolia, P, Perez Martinez, A, Rialland, F, Aljurf, M, Isgro, A, Toren, A, Bierings, M, Corbacioglu, S, Kalwak, K, Wachowiak J., Galimard J. -E., Dalissier A., Rihani R., AlSaedi H., Wynn R. F., Dalle J. -H., Peffault de Latour R., Sedlacek P., Balduzzi A., Schroeder T., Bodova I., Gonzalez Vicent M., Gruhn B., Hamladji R. -M., Krivan G., Patrick K., Sobkowiak-Sobierajska A., Stepensky P., Unal A., Amrolia P., Perez Martinez A., Rialland F., Aljurf M., Isgro A., Toren A., Bierings M., Corbacioglu S., Kalwak K., Wachowiak, J, Galimard, J, Dalissier, A, Rihani, R, Alsaedi, H, Wynn, R, Dalle, J, Peffault de Latour, R, Sedlacek, P, Balduzzi, A, Schroeder, T, Bodova, I, Gonzalez Vicent, M, Gruhn, B, Hamladji, R, Krivan, G, Patrick, K, Sobkowiak-Sobierajska, A, Stepensky, P, Unal, A, Amrolia, P, Perez Martinez, A, Rialland, F, Aljurf, M, Isgro, A, Toren, A, Bierings, M, Corbacioglu, S, Kalwak, K, Wachowiak J., Galimard J. -E., Dalissier A., Rihani R., AlSaedi H., Wynn R. F., Dalle J. -H., Peffault de Latour R., Sedlacek P., Balduzzi A., Schroeder T., Bodova I., Gonzalez Vicent M., Gruhn B., Hamladji R. -M., Krivan G., Patrick K., Sobkowiak-Sobierajska A., Stepensky P., Unal A., Amrolia P., Perez Martinez A., Rialland F., Aljurf M., Isgro A., Toren A., Bierings M., Corbacioglu S., and Kalwak K.

Abstract

This retrospective study evaluated 35 children (median age 5.2 years; range 0.4–18) with myelofibrosis (MF), including 33 with primary myelofibrosis and 2 with secondary myelofibrosis transplanted from matched sibling donor (MSD) (n = 17) or non-MSD (n = 18) between 2000 and 2022. Conditioning was usually chemotherapy-based (n = 33) and myeloablative (n = 32). Fifteen patients received bone marrow (BM), 14 haematopoietic cells (HC) from peripheral blood (PB), and 6 from cord blood (CB). Day +100 acute GvHD II–IV incidence was significantly lower after MSD-haematopoietic cell transplantation (MSD-HCT) than after non-MSD-HCT [18.8% (4.3–41.1) vs 58.8% (31–78.6); p = 0.01]. Six-year non-relapse mortality (NRM) was 18% (7.1–32.8), relapse incidence was 15.9% (5.6–30.9), progression-free survival (PFS) was 66.1% (47–79.7), GvHD-free relapse-free survival was 50% (30.6–66.7), and overall survival (OS) was 71.1% (51.4–84). Six-year PFS and OS were significantly higher after BM transplantation compared to HCT from other sources [85.1% (52.3–96.1) vs 50.8% (26.3–71), p = 0.03, and 90.9% (50.8–98.7) vs 54% (28.1–74.2), p = 0.01, respectively], whereas NRM was significantly lower [0% vs 32% (12.3–53.9); p = 0.02]. This first multicentre study on outcomes of allogeneic HCT in children with myelofibrosis proves feasibility and curative effect of transplantation in these children, suggests that bone marrow transplantation is associated with better outcomes, and indicates the need for further studies.

Published
2024

9. Outcomes of pediatric patients with therapy-related myeloid neoplasms

Author

Sharma, Akshay, Huang, Sujuan, Li, Ying, Brooke, Russell J., Ahmed, Ibrahim, Allewelt, Heather B., Amrolia, Persis, Bertaina, Alice, Bhatt, Neel S., Bierings, Marc B., Bies, Joshua, Brisset, Claire, Brondon, Jennifer E., Dahlberg, Ann, Dalle, Jean-Hugues, Eissa, Hesham, Fahd, Mony, Gassas, Adam, Gloude, Nicholas J., Goebel, W. Scott, Goeckerman, Erika S., Harris, Katherine, Ho, Richard, Hudspeth, Michelle P., Huo, Jeffrey S., Jacobsohn, David, Kasow, Kimberly A., Katsanis, Emmanuel, Kaviany, Saara, Keating, Amy K., Kernan, Nancy A., Ktena, Yiouli P., Lauhan, Colette R., López-Hernandez, Gerardo, Martin, Paul L., Myers, Kasiani C., Naik, Swati, Olaya-Vargas, Alberto, Onishi, Toshihiro, Radhi, Mohamed, Ramachandran, Shanti, Ramos, Kristie, Rangarajan, Hemalatha G., Roehrs, Philip A., Sampson, Megan E., Shaw, Peter J., Skiles, Jodi L., Somers, Katherine, Symons, Heather J., de Tersant, Marie, Uber, Allison N., Versluys, Birgitta, Cheng, Cheng, and Triplett, Brandon M.

Published
2021
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10. CAR T cells with dual targeting of CD19 and CD22 in pediatric and young adult patients with relapsed or refractory B cell acute lymphoblastic leukemia: a phase 1 trial

Author

Cordoba, Shaun, Onuoha, Shimobi, Thomas, Simon, Pignataro, Daniela Soriano, Hough, Rachael, Ghorashian, Sara, Vora, Ajay, Bonney, Denise, Veys, Paul, Rao, Kanchan, Lucchini, Giovanna, Chiesa, Robert, Chu, Jan, Clark, Liz, Fung, Mei Mei, Smith, Koval, Peticone, Carlotta, Al-Hajj, Muhammad, Baldan, Vania, Ferrari, Mathieu, Srivastava, Saket, Jha, Ram, Arce Vargas, Frederick, Duffy, Kevin, Day, William, Virgo, Paul, Wheeler, Lucy, Hancock, Jeremy, Farzaneh, Farzin, Domning, Sabine, Zhang, Yiyun, Khokhar, Nushmia Z., Peddareddigari, Vijay G. R., Wynn, Robert, Pule, Martin, and Amrolia, Persis J.

Published
2021
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12. Outcome of Non-hematological Autoimmunity After Hematopoietic Cell Transplantation in Children with Primary Immunodeficiency

Author

Lum, Su Han, Elfeky, Reem, Achini, Federica R., Margarit-Soler, Adriana, Cinicola, Bianca, Perez-Heras, Inigo, Nademi, Zohreh, Flood, Terry, Cheetham, Tim, Worth, Austen, Qasim, Waseem, Amin, Rakesh, Rao, Kanchan, Chiesa, Robert, Bredius, Robbert G. M., Amrolia, Persis, Abinun, Mario, Hambleton, Sophie, Veys, Paul, Gennery, Andrew R., Lankester, Arjan, and Slatter, Mary

Published
2021
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14. Activation priming and cytokine polyfunctionality modulate the enhanced functionality of low-affinity CD19 CAR T cells

Author

Michelozzi, I, Gomez-Castaneda, E, Pohle, R, Rodriguez, F, Sufi, J, Costa, P, Subramaniyam, M, Kirtsios, E, Eddaoudi, A, Wu, S, Guvenel, A, Fisher, J, Ghorashian, S, Pule, M, Tape, C, Castellano, S, Amrolia, P, Giustacchini, A, Michelozzi I. M., Gomez-Castaneda E., Pohle R. V. C., Rodriguez F. C., Sufi J., Costa P. P., Subramaniyam M., Kirtsios E., Eddaoudi A., Wu S. W., Guvenel A., Fisher J., Ghorashian S., Pule M. A., Tape C. J., Castellano S., Amrolia P. J., Giustacchini A., Michelozzi, I, Gomez-Castaneda, E, Pohle, R, Rodriguez, F, Sufi, J, Costa, P, Subramaniyam, M, Kirtsios, E, Eddaoudi, A, Wu, S, Guvenel, A, Fisher, J, Ghorashian, S, Pule, M, Tape, C, Castellano, S, Amrolia, P, Giustacchini, A, Michelozzi I. M., Gomez-Castaneda E., Pohle R. V. C., Rodriguez F. C., Sufi J., Costa P. P., Subramaniyam M., Kirtsios E., Eddaoudi A., Wu S. W., Guvenel A., Fisher J., Ghorashian S., Pule M. A., Tape C. J., Castellano S., Amrolia P. J., and Giustacchini A.

Abstract

We recently described a low-affinity second-generation CD19 chimeric antigen receptor (CAR) CAT that showed enhanced expansion, cytotoxicity, and antitumor efficacy compared with the high-affinity (FMC63-based) CAR used in tisagenlecleucel, in preclinical models. Furthermore, CAT demonstrated an excellent toxicity profile, enhanced in vivo expansion, and long-term persistence in a phase 1 clinical study. To understand the molecular mechanisms behind these properties of CAT CAR T cells, we performed a systematic in vitro characterization of the transcriptomic (RNA sequencing) and protein (cytometry by time of flight) changes occurring in T cells expressing low-affinity vs high-affinity CD19 CARs following stimulation with CD19-expressing cells. Our results show that CAT CAR T cells exhibit enhanced activation to CD19 stimulation and a distinct transcriptomic and protein profile, with increased activation and cytokine polyfunctionality compared with FMC63 CAR T cells. We demonstrate that the enhanced functionality of low-affinity CAT CAR T cells is a consequence of an antigen-dependent priming induced by residual CD19-expressing B cells present in the manufacture.

Published
2023

15. Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL treated with a low-affinity CD19 CAR

Author

Ghorashian, Sara, Kramer, Anne Marijn, Onuoha, Shimobi, Wright, Gary, Bartram, Jack, Richardson, Rachel, Albon, Sarah J., Casanovas-Company, Joan, Castro, Fernanda, Popova, Bilyana, Villanueva, Krystle, Yeung, Jenny, Vetharoy, Winston, Guvenel, Aleks, Wawrzyniecka, Patrycja A., Mekkaoui, Leila, Cheung, Gordon Weng-Kit, Pinner, Danielle, Chu, Jan, Lucchini, Giovanna, Silva, Juliana, Ciocarlie, Oana, Lazareva, Arina, Inglott, Sarah, Gilmour, Kimberly C., Ahsan, Gulrukh, Ferrari, Mathieu, Manzoor, Somayya, Champion, Kim, Brooks, Tony, Lopes, Andre, Hackshaw, Allan, Farzaneh, Farzin, Chiesa, Robert, Rao, Kanchan, Bonney, Denise, Samarasinghe, Sujith, Goulden, Nicholas, Vora, Ajay, Veys, Paul, Hough, Rachael, Wynn, Robert, Pule, Martin A., and Amrolia, Persis J.

Published
2019
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16. High transplant-related mortality associated with haematopoietic stem cell transplantation for paediatric therapy-related acute myeloid leukaemia (t-AML). A study on behalf of the United Kingdom Paediatric Blood and Bone Marrow Transplant Group

Author

Gassas, Adam, Sivaprakasam, Ponni, Cummins, Michelle, Breslin, Patricia, Patrick, Katharine, Slatter, Mary, Skinner, Roderick, Shenton, Geof, Gibson, Brenda, Lawson, Sarah, Petterson, Toni, Potter, Michael, James, Beki, Hough, Rachael, Hiwarkar, Prashant, Vora, Ajay, Veys, Paul, De La Fuente, Josu, Wynn, Robert, and Amrolia, Persis

Published
2018
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17. High-dimensional functional phenotyping of preclinical human CAR T cells using mass cytometry

Author

Michelozzi, I, Sufi, J, Adejumo, T, Amrolia, P, Tape, C, Giustacchini, A, Michelozzi I. M., Sufi J., Adejumo T. A., Amrolia P. J., Tape C. J., Giustacchini A., Michelozzi, I, Sufi, J, Adejumo, T, Amrolia, P, Tape, C, Giustacchini, A, Michelozzi I. M., Sufi J., Adejumo T. A., Amrolia P. J., Tape C. J., and Giustacchini A.

Abstract

Here, we present a comprehensive protocol for the generation and functional characterization of chimeric antigen receptor (CAR) T cells and their products by mass cytometry in a reproducible and scalable manner. We describe the production of CAR T cells from human peripheral blood mononuclear cells. We then detail a three-step staining protocol with metal-labeled antibodies and the subsequent mass cytometry analysis. This protocol allows simultaneous characterization of CAR T cell intracellular signaling, activation, proliferation, cytokine production, and phenotype in a single assay.

Published
2022

18. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., Yesilipek A., Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., and Yesilipek A.

Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long-term disease control or cure, but therapy-related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence-based interspeciality guidelines and recommendations.

Published
2022

22. Haematopoietic stem cell transplantation for severe autoimmune diseases in children : a review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini‐Gutzwiller, FR, Snowden, JA, Corbacioglu, S, Greco, R, Alexander, T, Snowden, J, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla‐Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries‐Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, HU, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, CH, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, MG, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, AP, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, and Yesilipek, A

Subjects
autoimmune diseases, haematopoietic stem cell transplantation, paediatric, ddc:610, paediatric, surgical procedures, operative, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, haematopoietic stem cell transplantation, 610 Medizin, autoimmune diseases, autoimmune disease, Hematology
Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (

Published
2022

23. CD19/CD22 targeting with cotransduced CAR T cells to prevent antigen-negative relapse after CAR T-cell therapy for B-cell ALL

Author

Ghorashian, Sara, Lucchini, Giovanna, Richardson, Rachel, Nguyen, Kyvi, Terris, Craig, Guvenel, Aleks, Oporto-Espuelas, Macarena, Yeung, Jenny, Pinner, Danielle, Chu, Jan, Williams, Lindsey, Ko, Ka-Yuk, Walding, Chloe, Watts, Kelly, Inglott, Sarah, Thomas, Rebecca, Connor, Christopher, Adams, Stuart, Gravett, Emma, Gilmour, Kimberly, Lal, Alka, Kunaseelan, Sangeetha, Popova, Bilyana, Lopes, Andre, Ngai, Yenting, Hackshaw, Allan, Kokalaki, Evangelia, Carulla, Milena Balasch, Mullanfiroze, Khushnuma, Lazareva, Arina, Pavasovic, Vesna, Rao, Anupama, Bartram, Jack, Vora, Ajay, Chiesa, Robert, Silva, Juliana, Rao, Kanchan, Bonney, Denise, Wynn, Robert, Pule, Martin, Hough, Rachael, and Amrolia, Persis J.

Abstract

•Dual-targeting CAR T cells cotransduced with CD19 and CD22 CARs were used to treat 12 patients with relapsed/refractory ALL with a 1-year EFS of 60%.•At a median follow-up of 8.7 months, there were no cases of antigen-negative relapse, suggesting this approach may prevent antigen escape.

Published
2024
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24. Impact of in Vivo Lymphodepletion on Outcome in Children with Nonmalignant Disorders Receiving Peripheral Blood Stem Cell Transplantation

Author

Ottaviano, G, Achini-Gutzwiller, F, Kalwak, K, Lanino, E, Faraci, M, Rao, K, Chiesa, R, Amrolia, P, Bonanomi, S, Rovelli, A, Veys, P, Lankester, A, Balduzzi, A, Lucchini, G, Ottaviano G., Achini-Gutzwiller F., Kalwak K., Lanino E., Faraci M., Rao K., Chiesa R., Amrolia P., Bonanomi S., Rovelli A., Veys P., Lankester A., Balduzzi A., Lucchini G., Ottaviano, G, Achini-Gutzwiller, F, Kalwak, K, Lanino, E, Faraci, M, Rao, K, Chiesa, R, Amrolia, P, Bonanomi, S, Rovelli, A, Veys, P, Lankester, A, Balduzzi, A, Lucchini, G, Ottaviano G., Achini-Gutzwiller F., Kalwak K., Lanino E., Faraci M., Rao K., Chiesa R., Amrolia P., Bonanomi S., Rovelli A., Veys P., Lankester A., Balduzzi A., and Lucchini G.

Abstract

Peripheral blood stem cell transplantation (PBSCT) with in vivo lymphodepletion can provide faster neutrophil recovery with limited risk of severe graft-versus-host disease (GVHD) in children with nonmalignant disorders (NMDs). We aimed to provide an historical comparison of these 2 strategies regarding the prevalence of GVHD, viral reactivation, timing of immune reconstitution, and final outcomes. Data on 98 children undergoing PBSCT were collected from 5 European pediatric transplantation centers. Only patients with NMDs receiving treosulfan or myeloablative busulfan conditioning and 9-10/10 HLA-matched transplant were included. The patients were divided into 2 groups according to in vivo lymphodepletion with antithymocyte globulin (ATG) or with alemtuzumab. We compared rates of acute and chronic GVHD; Epstein-Barr virus, cytomegalovirus, and adenovirus reactivation; chimerism; lymphocyte recovery; overall survival (OS) and event-free survival (EFS) between the 2 groups. The rate of severe acute GVHD (grade III-IV) was significantly higher in patients receiving ATG (26% vs 10% in alemtuzumab recipients; P < .05), whereas viral reactivations occurred with a similar rate in the 2 groups (alemtuzumab, 56%; ATG, 57%). Alemtuzumab was the major risk factor for delayed T cell immune reconstitution in the first 3 months after transplantation (odds ratio [OR], 6.0; 95% confidence interval [CI], 1.8 to 19; P < .005). Extended chronic GVHD, ADV reactivation, slower CD3+ cell recovery, and HLA-mismatch reduced the probability of survival. Infections were the main cause of mortality in our cohort, and delayed T cell recovery was significantly associated with mortality in multivariate analysis (OR, 12; 95% CI, 1.2 to 114; P < .05). Ultimately, no differences in OS and EFS survival were seen between the ATG and alemtuzumab groups. ATG and alemtuzumab showed similar impacts on outcomes of children undergoing PBSCT for NMDs. The 2 strategies of in vivo lymphodepletion sh

Published
2021

25. Non-Myeloablative Stem Cell Transplantation for Congenital Immunodeficiencies

Author

Gaspar, H. B., Amrolia, P., Hassan, A., Webb, D., Jones, A., Sturt, N., Vergani, G., Pagliuca, A., Mufti, G., Hadzic, N., Davies, G., Veys, P., Schlag, P. M., editor, Senn, H.-J., editor, Diehl, V., editor, Parkin, D. M., editor, Rajewsky, M. F., editor, Rubens, R., editor, Wannenmacher, M., editor, Rentchnik, P., editor, Oertel, Stephan H., editor, and Riess, Hanno, editor

Published
2002
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27. This title is unavailable for guests, please login to see more information.

Author

Locatelli, Franco, Thompson, A. A., Kwiatkowski, J. L., Porter, J. B., Thrasher, A. J., Hongeng, S., Sauer, M. G., Thuret, I., Lal, A., Algeri, M., Schneiderman, J., Olson, T. S., Carpenter, B., Amrolia, P. J., Anurathapan, U., Schambach, A., Chabannon, C., Schmidt, M., Labik, I., Elliot, H., Guo, R., Asmal, M., Colvin, R. A., Walters, M. C., Locatelli F. (ORCID:0000-0002-7976-3654), Locatelli, Franco, Thompson, A. A., Kwiatkowski, J. L., Porter, J. B., Thrasher, A. J., Hongeng, S., Sauer, M. G., Thuret, I., Lal, A., Algeri, M., Schneiderman, J., Olson, T. S., Carpenter, B., Amrolia, P. J., Anurathapan, U., Schambach, A., Chabannon, C., Schmidt, M., Labik, I., Elliot, H., Guo, R., Asmal, M., Colvin, R. A., Walters, M. C., and Locatelli F. (ORCID:0000-0002-7976-3654)

Published
2022

28. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini-Gutzwiller, F. R., Snowden, J. A., Corbacioglu, S., Greco, R., Alexander, T., Snowden, J., Badoglio, M., Labopin, M., Abinun, M., Apte, S., Arnold, R., Domenech, A., Brierley, C., Burman, J., Castilla-Llorente, C., Cooper, N., Daghia, G., Daikeler, T., del Papa, N., de Vries-Bouwstra, J., Farge, D., Finke, J., Hagglund, H., Hawkey, C., Henes, J., Hiepe, F., Jessop, H., Kiely, D., Kazmi, M., Kirgizov, K., Kramer, E., Mancardi, G., Marjanovic, Z., Martin, R., Martin, T., Ma, D., Moore, J., Miller, P., Muraro, P., Oliveira, M. -C., Polushin, A., Onida, F., Simoes, B., Puyade, M., Resnick, I., Ricart, E., Rovira, M., Saccardi, R., Saif, M., Sakellari, I., Sharrack, B., Snarski, E., Scherer, H. U., Sossa, C., Withers, B., Wulffraat, N., Zaccara, E., Amrolia, P., Ansari, M., Balduzzi, A., Dalassier, A., Dalle, J. -H., Diaz, C. H., Feuchtinger, T., Locatelli, Franco, Lucchini, G., Galimard, J. -E., Vincent, M. G., Handgretinger, R., Kleinschmidt, K., Lawitschka, A., Martinez, A. P., Peters, C., Rocha, V., Ruggeri, A., Sedlacek, P., Svec, P., Toporski, J., Yesilipek, A., Locatelli F. (ORCID:0000-0002-7976-3654), Achini-Gutzwiller, F. R., Snowden, J. A., Corbacioglu, S., Greco, R., Alexander, T., Snowden, J., Badoglio, M., Labopin, M., Abinun, M., Apte, S., Arnold, R., Domenech, A., Brierley, C., Burman, J., Castilla-Llorente, C., Cooper, N., Daghia, G., Daikeler, T., del Papa, N., de Vries-Bouwstra, J., Farge, D., Finke, J., Hagglund, H., Hawkey, C., Henes, J., Hiepe, F., Jessop, H., Kiely, D., Kazmi, M., Kirgizov, K., Kramer, E., Mancardi, G., Marjanovic, Z., Martin, R., Martin, T., Ma, D., Moore, J., Miller, P., Muraro, P., Oliveira, M. -C., Polushin, A., Onida, F., Simoes, B., Puyade, M., Resnick, I., Ricart, E., Rovira, M., Saccardi, R., Saif, M., Sakellari, I., Sharrack, B., Snarski, E., Scherer, H. U., Sossa, C., Withers, B., Wulffraat, N., Zaccara, E., Amrolia, P., Ansari, M., Balduzzi, A., Dalassier, A., Dalle, J. -H., Diaz, C. H., Feuchtinger, T., Locatelli, Franco, Lucchini, G., Galimard, J. -E., Vincent, M. G., Handgretinger, R., Kleinschmidt, K., Lawitschka, A., Martinez, A. P., Peters, C., Rocha, V., Ruggeri, A., Sedlacek, P., Svec, P., Toporski, J., Yesilipek, A., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long-term disease control or cure, but therapy-related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence-based interspeciality guidelines and recommendations.

Published
2022

29. S259: DUAL ANTIGEN TARGETING WITH CO-TRANSDUCED CD19/22 CAR T CELLS FOR RELAPSED/REFRACTORY ALL

Author

Ghorashian, S., primary, Lucchini, G., additional, Richardson, R., additional, Nguyen, K., additional, Terris, C., additional, Yeung, J., additional, Chu, J., additional, Williams, L., additional, Ko, K., additional, Walding, C., additional, Watts, K., additional, Inglott, S, additional, Adams, S., additional, Gravett, E., additional, Gilmour, K., additional, Lal, A., additional, Kunaseelan, S., additional, Popova, B., additional, Lopes, A., additional, Ngai, Y., additional, Kokalaki, E., additional, Rao, K., additional, Chiesa, R., additional, Silva, J., additional, Mullanfiroze, K, additional, Lazareva, A., additional, Bonney, D., additional, Wynn, R, additional, Pule, M., additional, Hough, R., additional, and Amrolia, P., additional

Published
2022
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32. The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing

Author

de la Fuente, J, Gluckman, E, Makani, J, Telfer, P, Faulkner, L, Corbacioglu, S, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Hereda Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Gonzalez Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Perez Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, de la Fuente J., Gluckman E., Makani J., Telfer P., Faulkner L., Corbacioglu S., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Hereda Diaz C., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Gonzalez Vincent M., Handgretinger R., Kleinschmidt K., Lawitschka A., Perez Martinez A., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., Yesilipek A., de la Fuente, J, Gluckman, E, Makani, J, Telfer, P, Faulkner, L, Corbacioglu, S, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Hereda Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Gonzalez Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Perez Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, de la Fuente J., Gluckman E., Makani J., Telfer P., Faulkner L., Corbacioglu S., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Hereda Diaz C., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Gonzalez Vincent M., Handgretinger R., Kleinschmidt K., Lawitschka A., Perez Martinez A., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., and Yesilipek A.

Abstract

Sickle cell disease is one of the most common, life-threatening, non-communicable diseases in the world and a major public health problem. Following the implementation of simple preventive and therapeutic modalities, infant mortality has almost been abolished in high-income countries, but only a small amount of progress has been made in improving survival in adulthood. Progressive end-organ damage, partly related to a systemic vasculopathy, is increasingly recognised. With the availability of a variety of novel disease-modifying drugs, gene addition and gene editing strategies, matched sibling donor haematopoietic stem cell transplantation (HSCT) in children (offering an overall survival rate of 95% and an event-free survival rate of 92%), and encouraging outcomes after alternative donor HSCT, the new challenge is to risk stratify patients, revise transplantation indications, and define the best therapeutic approach for each patient. The ultimate challenge will be to enable these advances in low-income and middle-income countries, where disease prevalence is highest and where innovative strategies are most needed.

Published
2020

38. T-cell replete cord transplants give superior outcomes in high-risk and relapsed/refractory pediatric myeloid malignancy

Author

Horgan, Claire, Mullanfiroze, Khushnuma, Rauthan, Archana, Patrick, Katharine, Butt, Naeem Akram, Mirci-Danicar, Oana, O’Connor, Olya, Furness, Caroline, Deshpande, Akshay, Lawson, Sarah, Broderick, Valerie, Evans, Pamela, Gibson, Brenda, Roberts, Wing, Ali, Salah, Galani, Sevasti, Kirkwood, Amy A., Jovanovic, Jelena, Dillon, Richard, Virgo, Paul, James, Beki, Rao, Kanchan, Amrolia, Persis J., and Wynn, Robert F.

Abstract

•Compared with other cell sources, TRCB transplant results in improved disease-free survival and relapse risk in pediatric AML/MDS.•Compared with other cell sources, cord-transplant cures with less chronic GvHD and particularly improves GvHD-free, relapse-free survival.

Published
2023
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42. Dual-targeting CAR T cells for B-cell acute lymphoblastic leukemia and B-cell non-Hodgkin lymphoma

Author

de Oliveira Canedo, Gustavo, Roddie, Claire, and Amrolia, Persis J.

Abstract

Relapse after CD19-directed chimeric antigen receptor (CAR) T-cell therapy remains a major challenge in B-cell acute lymphoblastic leukemia (ALL) and B-cell non-Hodgkin lymphoma (B-NHL). One of the main strategies to avoid CD19-negative relapse has been the development of dual CAR T cells targeting CD19 and an additional target, such as CD22 or CD20. Different methods have been used to achieve this, including coadministration of 2 products targeting 1 single antigen, cotransduction of autologous T cells, use of a bicistronic vector, or the development of bivalent CARs. Phase 1 and 2 trials across all manufacturing strategies have shown this to be a safe approach with equivalent remission rates and initial product expansion. CAR T-cell persistence remains a significant issue, with the majority of relapses being antigen-positive after CAR T-cell infusion. Further, despite adding a second antigen, antigen-negative relapses have not yet been eliminated. This review summarizes the state of the art with dual-targeting CAR T cells for B-cell ALL and B-NHL, the challenges encountered, and possible next steps to overcome them.

Published
2025
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