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1. Pre-Transplant Marital Status and Hematopoietic Cell Transplantation Outcomes.

Author

Ulrickson, M, Verdonck, L, Wirk, B, Wood, W, Yared, J, Saber, W, Tay, J, Beattie, S, Bredeson, C, Brazauskas, R, He, N, Ahmed, I, Aljurf, M, Askar, M, Atsuta, Y, Badawy, S, Barata, A, Beitinjaneh, A, Bhatt, N, Buchbinder, D, Cerny, J, Ciurea, S, DSouza, A, Dalal, J, Farhadfar, N, Freytes, C, Ganguly, S, Gergis, U, Gerull, S, Lazarus, H, Hahn, T, Hong, S, Inamoto, Y, Khera, N, Kindwall-Keller, T, Kamble, R, Knight, J, Koleva, Y, Kumar, A, Kwok, J, Murthy, H, Olsson, R, Angel Diaz-Perez, M, Rizzieri, D, Seo, S, Chhabra, S, Schoemans, H, Schouten, H, Steinberg, A, Sullivan, K, Szer, J, and Szwajcer, D

Subjects
Hematopoietic cell transplantation, graft-versus-host disease, marital status, overall survival, registries, Graft vs Host Disease, Hematopoietic Stem Cell Transplantation, Humans, Marital Status, Quality of Life
Abstract

BACKGROUND: Evidence about the impact of marital status before hematopoietic cell transplantation (hct) on outcomes after hct is conflicting. METHODS: We identified patients 40 years of age and older within the Center for International Blood and Marrow Transplant Research registry who underwent hct between January 2008 and December 2015. Marital status before hct was declared as one of: married or living with a partner, single (never married), separated or divorced, and widowed. We performed a multivariable analysis to determine the association of marital status with outcomes after hct. RESULTS: We identified 10,226 allogeneic and 5714 autologous hct cases with, respectively, a median follow-up of 37 months (range: 1-102 months) and 40 months (range: 1-106 months). No association between marital status and overall survival was observed in either the allogeneic (p = 0.58) or autologous (p = 0.17) setting. However, marital status was associated with grades 2-4 acute graft-versus-host disease (gvhd), p < 0.001, and chronic gvhd, p = 0.04. The risk of grades 2-4 acute gvhd was increased in separated compared with married patients [hazard ratio (hr): 1.13; 95% confidence interval (ci): 1.03 to 1.24], and single patients had a reduced risk of grades 2-4 acute gvhd (hr: 0.87; 95% ci: 0.77 to 0.98). The risk of chronic gvhd was lower in widowed compared with married patients (hr: 0.82; 95% ci: 0.67 to 0.99). CONCLUSIONS: Overall survival after hct is not influenced by marital status, but associations were evident between marital status and grades 2-4 acute and chronic gvhd. To better appreciate the effects of marital status and social support, future research should consider using validated scales to measure social support and patient and caregiver reports of caregiver commitment, and to assess health-related quality of life together with health care utilization.

Published
2020

2. 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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3. Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

Author

Vallée, T, Glasmacher, J, Buchner, H, Arkwright, P, Behrends, U, Bondarenko, A, Browning, M, Buchbinder, D, Cattoni, A, Chernyshova, L, Ciznar, P, Cole, T, Czogala, W, Dueckers, G, Edgar, J, Erbey, F, Fasth, A, Ferrua, F, Formankova, R, Gambineri, E, Gennery, A, Goldman, F, Gonzalez-Granado, L, Heilmann, C, Heiskanen-Kosma, T, Juntti, H, Kainulainen, L, Kanegane, H, Karaca, N, Sebnem Kilic, S, Klein, C, Koltan, S, Kondratenko, I, Meyts, I, Nasrullayeva, G, Notarangelo, L, Pasic, S, Pellier, I, Pignata, C, Misbah, S, Schulz, A, Segundo, G, Shcherbina, A, Slatter, M, Sokolic, R, Soler-Palacin, P, Stepensky, P, van Montfrans, J, Ryhänen, S, Wolska-Kuśnierz, B, Ziegler, J, Zhao, X, Aiuti, A, Ochs, H, Albert, M, Vallée, Tanja C, Glasmacher, Jannik S, Buchner, Hannes, Arkwright, Peter D, Behrends, Uta, Bondarenko, Anastasia, Browning, Michael J, Buchbinder, David K, Cattoni, Alessandro, Chernyshova, Liudmyla, Ciznar, Peter, Cole, Theresa, Czogala, Wojciech, Dueckers, Gregor, Edgar, John David M, Erbey, Fatih, Fasth, Anders, Ferrua, Francesca, Formankova, Renata, Gambineri, Eleonora, Gennery, Andrew R, Goldman, Frederick D, Gonzalez-Granado, Luis Ignacio, Heilmann, Carsten, Heiskanen-Kosma, Tarja, Juntti, Hanna, Kainulainen, Leena, Kanegane, Hirokazu, Karaca, Neslihan E., Sebnem Kilic, Sara, Klein, Christoph, Koltan, Sylwia, Kondratenko, Irina, Meyts, Isabelle, Nasrullayeva, Gulnara M, Notarangelo, Lucia Dora, Pasic, Srdjan, Pellier, Isabelle, Pignata, Claudio, Misbah, Siraj Ahmed, Schulz, Ansgar S, Segundo, Gesmar RS, Shcherbina, Anna, Slatter, Mary A, Sokolic, Robert, Soler-Palacin, Pere, Stepensky, Polina, van Montfrans, Joris M., Ryhänen, Samppa, Wolska-Kuśnierz, Beata, Ziegler, John B, Zhao, Xiaodong, Aiuti, Alessandro, Ochs, Hans D, Albert, Michael H, Vallée, T, Glasmacher, J, Buchner, H, Arkwright, P, Behrends, U, Bondarenko, A, Browning, M, Buchbinder, D, Cattoni, A, Chernyshova, L, Ciznar, P, Cole, T, Czogala, W, Dueckers, G, Edgar, J, Erbey, F, Fasth, A, Ferrua, F, Formankova, R, Gambineri, E, Gennery, A, Goldman, F, Gonzalez-Granado, L, Heilmann, C, Heiskanen-Kosma, T, Juntti, H, Kainulainen, L, Kanegane, H, Karaca, N, Sebnem Kilic, S, Klein, C, Koltan, S, Kondratenko, I, Meyts, I, Nasrullayeva, G, Notarangelo, L, Pasic, S, Pellier, I, Pignata, C, Misbah, S, Schulz, A, Segundo, G, Shcherbina, A, Slatter, M, Sokolic, R, Soler-Palacin, P, Stepensky, P, van Montfrans, J, Ryhänen, S, Wolska-Kuśnierz, B, Ziegler, J, Zhao, X, Aiuti, A, Ochs, H, Albert, M, Vallée, Tanja C, Glasmacher, Jannik S, Buchner, Hannes, Arkwright, Peter D, Behrends, Uta, Bondarenko, Anastasia, Browning, Michael J, Buchbinder, David K, Cattoni, Alessandro, Chernyshova, Liudmyla, Ciznar, Peter, Cole, Theresa, Czogala, Wojciech, Dueckers, Gregor, Edgar, John David M, Erbey, Fatih, Fasth, Anders, Ferrua, Francesca, Formankova, Renata, Gambineri, Eleonora, Gennery, Andrew R, Goldman, Frederick D, Gonzalez-Granado, Luis Ignacio, Heilmann, Carsten, Heiskanen-Kosma, Tarja, Juntti, Hanna, Kainulainen, Leena, Kanegane, Hirokazu, Karaca, Neslihan E., Sebnem Kilic, Sara, Klein, Christoph, Koltan, Sylwia, Kondratenko, Irina, Meyts, Isabelle, Nasrullayeva, Gulnara M, Notarangelo, Lucia Dora, Pasic, Srdjan, Pellier, Isabelle, Pignata, Claudio, Misbah, Siraj Ahmed, Schulz, Ansgar S, Segundo, Gesmar RS, Shcherbina, Anna, Slatter, Mary A, Sokolic, Robert, Soler-Palacin, Pere, Stepensky, Polina, van Montfrans, Joris M., Ryhänen, Samppa, Wolska-Kuśnierz, Beata, Ziegler, John B, Zhao, Xiaodong, Aiuti, Alessandro, Ochs, Hans D, and Albert, Michael H

Abstract

Wiskott-Aldrich syndrome (WAS) is a multifaceted monogenic disorder with a broad disease spectrum and variable disease severity and a variety of treatment options including allogeneic hematopoietic stem cell transplantation (HSCT) and gene therapy (GT). No reliable biomarker exists to predict disease course and outcome for individual patients. A total of 577 patients with a WAS variant from 26 countries and a median follow-up of 8.9 years (range, 0.3-71.1), totaling 6118 patient-years, were included in this international retrospective study. Overall survival (OS) of the cohort (censored at HSCT or GT) was 82% (95% confidence interval, 78-87) at age 15 years and 70% (61-80) at 30 years. The type of variant was predictive of outcome: patients with a missense variant in exons 1 or 2 or with the intronic hot spot variant c.559+5G>A (class I variants) had a 15-year OS of 93% (89-98) and a 30-year OS of 91% (86-97), compared with 71% (62-81) and 48% (34-68) in patients with any other variant (class II; P < .0001). The cumulative incidence rates of disease-related complications such as severe bleeding (P = .007), life-threatening infection (P < .0001), and autoimmunity (P = .004) occurred significantly later in patients with a class I variant. The cumulative incidence of malignancy (P = .6) was not different between classes I and II. It confirms the spectrum of disease severity and quantifies the risk for specific disease-related complications. The class of the variant is a biomarker to predict the outcome for patients with WAS.

Published
2024

4. Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy

Author

Buchbinder D, Nugent D, and Hsieh L

Subjects
thrombocytopenia, chronic, thrombopoietin, childhood, Therapeutics. Pharmacology, RM1-950
Abstract

David Buchbinder, Diane Nugent, Loan Hsieh Division of Hematology, CHOC Children’s Hospital and UC Irvine Medical Center, CA, USA Abstract: Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia. In approximately one-third of cases, the duration of thrombocytopenia will extend beyond 12 months consistent with a diagnosis of chronic ITP. Minor bleeding manifestations are common in chronic ITP while severe or life-threatening bleeding complications are uncommon. Moreover, spontaneous resolution occurs in the majority of children with chronic ITP necessitating treatment in only those children with ongoing bleeding manifestations or impairment in health-related quality of life (HRQOL). The characterization of thrombopoietin (TPO) and remarkable advancements in our understanding of the pathophysiology of ITP has led to the development of a new class of agents, the TPO-receptor agonists that have documented efficacy in the amelioration of thrombocytopenia and bleeding manifestations in chronic ITP. Romiplostim is a second-generation TPO-receptor agonist that has undergone limited evaluation in the treatment of chronic ITP in children. Evolving data suggest that romiplostim may be a safe and effective agent in the treatment of chronic ITP in children. Additional data are needed to confirm its ability to increase platelet counts, decrease bleeding manifestation, and improve the HRQOL of children and caregivers impacted by chronic ITP. Keywords: thrombocytopenia, chronic, thrombopoietin, childhood

Published
2017

5. Wiskott–Aldrich syndrome: diagnosis, current management, and emerging treatments

Author

Buchbinder D, Nugent DJ, and Filipovich AH

Subjects
Medicine (General), R5-920, Genetics, QH426-470
Abstract

David Buchbinder,1 Diane J Nugent,1 Alexandra H Fillipovich2 1Division of Hematology, Children's Hospital of Orange County, Orange, CA, USA; 2Division of Immunology, Cincinnati Children's Hospital, Cincinnati, OH, USA Abstract: Wiskott–Aldrich syndrome (WAS) is a rare X-linked primary immunodeficiency disorder characterized by the triad of eczema, thrombocytopenia, and severe and often recurrent infections. Despite the rarity of this disorder, our understanding of the molecular and cellular pathogenesis of WAS has continued to increase. Advances in the use of diagnostic tools, the provision of supportive care, and improvements in allogeneic hematopoietic stem cell transplantation have significantly reduced the morbidity and mortality associated with this disorder. Exciting advancements in the care of patients with WAS have also occurred, including the successful application of autologous gene-modified hematopoietic stem cell transplantation. Keywords: primary immunodeficiency, Wiskott–Aldrich syndrome, hematopoietic stem cell transplantation, gene therapy

Published
2014

7. Disease Progression of WHIM Syndrome in an International Cohort of 66 Pediatric and Adult Patients

Author

Geier, CB, Ellison, M, Cruz, R, Pawar, S, Leiss-Piller, A, Zmajkovicova, K, McNulty, SM, Yilmaz, M, Evans, MO, Gordon, S, Ujhazi, B, Wiest, I, Abolhassani, H, Aghamohammadi, A, Barmettler, S, Bhar, S, Bondarenko, A, Bolyard, AA, Buchbinder, D, Cada, M, Cavieres, M, Connelly, JA, Dale, DC, Deordieva, E, Dorsey, MJ, Drysdale, SB, Ehl, S, Elfeky, R, Fioredda, F, Firkin, F, Forster-Waldl, E, Geng, B, Goda, V, Gonzalez-Granado, L, Grunebaum, E, Grzesk, E, Henrickson, SE, Hilfanova, A, Hiwatari, M, Imai, C, Ip, W, Jyonouchi, S, Kanegane, H, Kawahara, Y, Khojah, AM, Kim, VH-D, Kojic, M, Koltan, S, Krivan, G, Langguth, D, Lau, Y-L, Leung, D, Miano, M, Mersyanova, I, Mousallem, T, Muskat, M, Naoum, FA, Noronha, SA, Ouederni, M, Ozono, S, Richmond, GW, Sakovich, I, Salzer, U, Schuetz, C, Seeborg, FO, Sharapova, SO, Sockel, K, Volokha, A, von Bonin, M, Warnatz, K, Wegehaupt, O, Weinberg, GA, Wong, K-J, Worth, A, Yu, H, Zharankova, Y, Zhao, X, Devlin, L, Badarau, A, Csomos, K, Keszei, M, Pereira, J, Taveras, AG, Beaussant-Cohen, SL, Ong, M-S, Shcherbina, A, Walter, JE, Geier, CB, Ellison, M, Cruz, R, Pawar, S, Leiss-Piller, A, Zmajkovicova, K, McNulty, SM, Yilmaz, M, Evans, MO, Gordon, S, Ujhazi, B, Wiest, I, Abolhassani, H, Aghamohammadi, A, Barmettler, S, Bhar, S, Bondarenko, A, Bolyard, AA, Buchbinder, D, Cada, M, Cavieres, M, Connelly, JA, Dale, DC, Deordieva, E, Dorsey, MJ, Drysdale, SB, Ehl, S, Elfeky, R, Fioredda, F, Firkin, F, Forster-Waldl, E, Geng, B, Goda, V, Gonzalez-Granado, L, Grunebaum, E, Grzesk, E, Henrickson, SE, Hilfanova, A, Hiwatari, M, Imai, C, Ip, W, Jyonouchi, S, Kanegane, H, Kawahara, Y, Khojah, AM, Kim, VH-D, Kojic, M, Koltan, S, Krivan, G, Langguth, D, Lau, Y-L, Leung, D, Miano, M, Mersyanova, I, Mousallem, T, Muskat, M, Naoum, FA, Noronha, SA, Ouederni, M, Ozono, S, Richmond, GW, Sakovich, I, Salzer, U, Schuetz, C, Seeborg, FO, Sharapova, SO, Sockel, K, Volokha, A, von Bonin, M, Warnatz, K, Wegehaupt, O, Weinberg, GA, Wong, K-J, Worth, A, Yu, H, Zharankova, Y, Zhao, X, Devlin, L, Badarau, A, Csomos, K, Keszei, M, Pereira, J, Taveras, AG, Beaussant-Cohen, SL, Ong, M-S, Shcherbina, A, and Walter, JE

Abstract

Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome (WS) is a combined immunodeficiency caused by gain-of-function mutations in the C-X-C chemokine receptor type 4 (CXCR4) gene. We characterize a unique international cohort of 66 patients, including 57 (86%) cases previously unreported, with variable clinical phenotypes. Of 17 distinct CXCR4 genetic variants within our cohort, 11 were novel pathogenic variants affecting 15 individuals (23%). All variants affect the same CXCR4 region and impair CXCR4 internalization resulting in hyperactive signaling. The median age of diagnosis in our cohort (5.5 years) indicates WHIM syndrome can commonly present in childhood, although some patients are not diagnosed until adulthood. The prevalence and mean age of recognition and/or onset of clinical manifestations within our cohort were infections 88%/1.6 years, neutropenia 98%/3.8 years, lymphopenia 88%/5.0 years, and warts 40%/12.1 years. However, we report greater prevalence and variety of autoimmune complications of WHIM syndrome (21.2%) than reported previously. Patients with versus without family history of WHIM syndrome were diagnosed earlier (22%, average age 1.3 years versus 78%, average age 5 years, respectively). Patients with a family history of WHIM syndrome also received earlier treatment, experienced less hospitalization, and had less end-organ damage. This observation reinforces previous reports that early treatment for WHIM syndrome improves outcomes. Only one patient died; death was attributed to complications of hematopoietic stem cell transplantation. The variable expressivity of WHIM syndrome in pediatric patients delays their diagnosis and therapy. Early-onset bacterial infections with severe neutropenia and/or lymphopenia should prompt genetic testing for WHIM syndrome, even in the absence of warts.

Published
2022

10. Pre-transplant marital status and hematopoietic cell transplantation outcomes

Author

Tay, J., Beattie, S., Bredeson, C., Brazauskas, R., He, N., Ahmed, I. A., Aljurf, M., Askar, M., Atsuta, Y., Badawy, S., Barata, A., Beitinjaneh, A. M., Bhatt, N. S., Buchbinder, D., Cerny, J., Ciurea, S., D'Souza, A., Dalal, J., Farhadfar, N., Freytes, C. O., Ganguly, S., Gergis, U., Gerull, S., Lazarus, H. M., Hahn, T., Hong, S., Inamoto, Y., Khera, N., Kindwall-Keller, T., Kamble, R. T., Knight, J. M., Koleva, Y. N., Kumar, A., Kwok, J., Murthy, H. S., Olsson, R. F., Angel Diaz-Perez, M., Rizzieri, D., Seo, S., Chhabra, S., Schoemans, H., Schouten, H. C., Steinberg, A., Sullivan, K. M., Szer, J., Szwajcer, D., Ulrickson, M. L., Verdonck, L. F., Wirk, B., Wood, W. A., Yared, J. A., Saber, W., Tay, J., Beattie, S., Bredeson, C., Brazauskas, R., He, N., Ahmed, I. A., Aljurf, M., Askar, M., Atsuta, Y., Badawy, S., Barata, A., Beitinjaneh, A. M., Bhatt, N. S., Buchbinder, D., Cerny, J., Ciurea, S., D'Souza, A., Dalal, J., Farhadfar, N., Freytes, C. O., Ganguly, S., Gergis, U., Gerull, S., Lazarus, H. M., Hahn, T., Hong, S., Inamoto, Y., Khera, N., Kindwall-Keller, T., Kamble, R. T., Knight, J. M., Koleva, Y. N., Kumar, A., Kwok, J., Murthy, H. S., Olsson, R. F., Angel Diaz-Perez, M., Rizzieri, D., Seo, S., Chhabra, S., Schoemans, H., Schouten, H. C., Steinberg, A., Sullivan, K. M., Szer, J., Szwajcer, D., Ulrickson, M. L., Verdonck, L. F., Wirk, B., Wood, W. A., Yared, J. A., and Saber, W.

Abstract

Background Evidence about the impact of marital status before hematopoietic cell transplantation (HCT) on outcomes after HCT is conflicting. Methods We identified patients 40 years of age and older within the Center for International Blood and Marrow Transplant Research registry who underwent HCT between January 2008 and December 2015. Marital status before HCT was declared as one of: married or living with a partner, single (never married), separated or divorced, and widowed. We performed a multivariable analysis to determine the association of marital status with outcomes after HCT. Results We identified 10,226 allogeneic and 5714 autologous HCT cases with, respectively, a median follow-up of 37 months (range: 1-102 months) and 40 months (range: 1-106 months). No association between marital status and overall survival was observed in either the allogeneic (p = 0.58) or autologous (p = 0.17) setting. However, marital status was associated with grades 2-4 acute graft-versus-host disease (GVHD), p < 0.001, and chronic GVHD, p = 0.04. The risk of grades 2-4 acute GVHD was increased in separated compared with married patients [hazard ratio (FIR): 1.13; 95% confidence interval (CI): 1.03 to 1.24], and single patients had a reduced risk of grades 2-4 acute GVHD (FIR: 0.87; 95% CI: 0.77 to 0.98). The risk of chronic GVHD was lower in widowed compared with married patients (FIR: 0.82; 95% CI: 0.67 to 0.99). Conclusions Overall survival after HCT is not influenced by marital status, but associations were evident between marital status and grades 2-4 acute and chronic GVHD. To better appreciate the effects of marital status and social support, future research should consider using validated scales to measure social support and patient and caregiver reports of caregiver commitment, and to assess health-related quality of life together with health care utilization.

Published
2020
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11. Pre-transplant marital status and hematopoietic cell transplantation outcomes

Author

Tay, J, Beattie, S, Bredeson, C, Brazauskas, R, He, N, Ahmed, IA, Aljurf, M, Askar, M, Atsuta, Y, Badawy, S, Barata, A, Beitinjaneh, AM, Bhatt, NS, Buchbinder, D, Cerny, J, Ciurea, S, D'Souza, A, Dalal, J, Farhadfar, N, Freytes, CO, Ganguly, S, Gergis, U, Gerull, S, Lazarus, HM, Hahn, T, Hong, S, Inamoto, Y, Khera, N, Kindwall-Keller, T, Kamble, RT, Knight, JM, Koleva, YN, Kumar, A, Kwok, J, Murthy, HS, Olsson, RF, Angel Diaz-Perez, M, Rizzieri, D, Seo, S, Chhabra, S, Schoemans, H, Schouten, HC, Steinberg, A, Sullivan, KM, Szer, J, Szwajcer, D, Ulrickson, ML, Verdonck, LF, Wirk, B, Wood, WA, Yared, JA, Saber, W, Tay, J, Beattie, S, Bredeson, C, Brazauskas, R, He, N, Ahmed, IA, Aljurf, M, Askar, M, Atsuta, Y, Badawy, S, Barata, A, Beitinjaneh, AM, Bhatt, NS, Buchbinder, D, Cerny, J, Ciurea, S, D'Souza, A, Dalal, J, Farhadfar, N, Freytes, CO, Ganguly, S, Gergis, U, Gerull, S, Lazarus, HM, Hahn, T, Hong, S, Inamoto, Y, Khera, N, Kindwall-Keller, T, Kamble, RT, Knight, JM, Koleva, YN, Kumar, A, Kwok, J, Murthy, HS, Olsson, RF, Angel Diaz-Perez, M, Rizzieri, D, Seo, S, Chhabra, S, Schoemans, H, Schouten, HC, Steinberg, A, Sullivan, KM, Szer, J, Szwajcer, D, Ulrickson, ML, Verdonck, LF, Wirk, B, Wood, WA, Yared, JA, and Saber, W

Abstract

BACKGROUND: Evidence about the impact of marital status before hematopoietic cell transplantation (hct) on outcomes after hct is conflicting. METHODS: We identified patients 40 years of age and older within the Center for International Blood and Marrow Transplant Research registry who underwent hct between January 2008 and December 2015. Marital status before hct was declared as one of: married or living with a partner, single (never married), separated or divorced, and widowed. We performed a multivariable analysis to determine the association of marital status with outcomes after hct. RESULTS: We identified 10,226 allogeneic and 5714 autologous hct cases with, respectively, a median follow-up of 37 months (range: 1-102 months) and 40 months (range: 1-106 months). No association between marital status and overall survival was observed in either the allogeneic (p = 0.58) or autologous (p = 0.17) setting. However, marital status was associated with grades 2-4 acute graft-versus-host disease (gvhd), p < 0.001, and chronic gvhd, p = 0.04. The risk of grades 2-4 acute gvhd was increased in separated compared with married patients [hazard ratio (hr): 1.13; 95% confidence interval (ci): 1.03 to 1.24], and single patients had a reduced risk of grades 2-4 acute gvhd (hr: 0.87; 95% ci: 0.77 to 0.98). The risk of chronic gvhd was lower in widowed compared with married patients (hr: 0.82; 95% ci: 0.67 to 0.99). CONCLUSIONS: Overall survival after hct is not influenced by marital status, but associations were evident between marital status and grades 2-4 acute and chronic gvhd. To better appreciate the effects of marital status and social support, future research should consider using validated scales to measure social support and patient and caregiver reports of caregiver commitment, and to assess health-related quality of life together with health care utilization.

Published
2020

12. Pre-Transplant Marital Status and Hematopoietic Cell Transplantation Outcomes

Author

Tay, J., primary, Beattie, S., additional, Bredeson, C., additional, Brazauskas, R., additional, He, N., additional, Ahmed, I. A., additional, Aljurf, M., additional, Askar, M., additional, Atsuta, Y., additional, Badawy, S., additional, Barata, A., additional, Beitinjaneh, A. M., additional, Bhatt, N. S., additional, Buchbinder, D., additional, Cerny, J., additional, Ciurea, S., additional, D’Souza, A., additional, Dalal, J., additional, Farhadfar, N., additional, Freytes, C. O., additional, Ganguly, S., additional, Gergis, U., additional, Gerull, S., additional, Lazarus, H. M., additional, Hahn, T., additional, Hong, S., additional, Inamoto, Y., additional, Khera, N., additional, Kindwall-Keller, T., additional, Kamble, R. T., additional, Knight, J. M., additional, Koleva, Y. N., additional, Kumar, A., additional, Kwok, J., additional, Murthy, H. S., additional, Olsson, R. F., additional, Angel Diaz-Perez, M., additional, Rizzieri, D., additional, Seo, S., additional, Chhabra, S., additional, Schoemans, H., additional, Schouten, H. C., additional, Steinberg, A., additional, Sullivan, K. M., additional, Szer, J., additional, Szwajcer, D., additional, Ulrickson, M. L., additional, Verdonck, L. F., additional, Wirk, B., additional, Wood, W. A., additional, Yared, J. A., additional, and Saber, W., additional

Published
2020
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16. Outcomes and Treatment Strategies for Autoimmunity and Hyperinflammation in Patients with RAG Deficiency

Author

Farmer, J.R. Foldvari, Z. Ujhazi, B. De Ravin, S.S. Chen, K. Bleesing, J.J.H. Schuetz, C. Al-Herz, W. Abraham, R.S. Joshi, A.Y. Costa-Carvalho, B.T. Buchbinder, D. Booth, C. Reiff, A. Ferguson, P.J. Aghamohammadi, A. Abolhassani, H. Puck, J.M. Adeli, M. Cancrini, C. Palma, P. Bertaina, A. Locatelli, F. Di Matteo, G. Geha, R.S. Kanariou, M.G. Lycopoulou, L. Tzanoudaki, M. Sleasman, J.W. Parikh, S. Pinero, G. Fischer, B.M. Dbaibo, G. Unal, E. Patiroglu, T. Karakukcu, M. Al-Saad, K.K. Dilley, M.A. Pai, S.-Y. Dutmer, C.M. Gelfand, E.W. Geier, C.B. Eibl, M.M. Wolf, H.M. Henderson, L.A. Hazen, M.M. Bonfim, C. Wolska-Kuśnierz, B. Butte, M.J. Hernandez, J.D. Nicholas, S.K. Stepensky, P. Chandrakasan, S. Miano, M. Westermann-Clark, E. Goda, V. Kriván, G. Holland, S.M. Fadugba, O. Henrickson, S.E. Ozen, A. Karakoc-Aydiner, E. Baris, S. Kiykim, A. Bredius, R. Hoeger, B. Boztug, K. Pashchenko, O. Neven, B. Moshous, D. de Villartay, J.-P. Bousfiha, A.A. Hill, H.R. Notarangelo, L.D. Walter, J.E.

Subjects
hemic and lymphatic diseases
Abstract

Background: Although autoimmunity and hyperinflammation secondary to recombination activating gene (RAG) deficiency have been associated with delayed diagnosis and even death, our current understanding is limited primarily to small case series. Objective: Understand the frequency, severity, and treatment responsiveness of autoimmunity and hyperinflammation in RAG deficiency. Methods: In reviewing the literature and our own database, we identified 85 patients with RAG deficiency, reported between 2001 and 2016, and compiled the largest case series to date of 63 patients with prominent autoimmune and/or hyperinflammatory pathology. Results: Diagnosis of RAG deficiency was delayed a median of 5 years from the first clinical signs of immune dysregulation. Most patients (55.6%) presented with more than 1 autoimmune or hyperinflammatory complication, with the most common etiologies being cytopenias (84.1%), granulomas (23.8%), and inflammatory skin disorders (19.0%). Infections, including live viral vaccinations, closely preceded the onset of autoimmunity in 28.6% of cases. Autoimmune cytopenias had early onset (median, 1.9, 2.1, and 2.6 years for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively) and were refractory to intravenous immunoglobulin, steroids, and rituximab in most cases (64.7%, 73.7%, and 71.4% for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively). Evans syndrome specifically was associated with lack of response to first-line therapy. Treatment-refractory autoimmunity/hyperinflammation prompted hematopoietic stem cell transplantation in 20 patients. Conclusions: Autoimmunity/hyperinflammation can be a presenting sign of RAG deficiency and should prompt further evaluation. Multilineage cytopenias are often refractory to immunosuppressive treatment and may require hematopoietic cell transplantation for definitive management. © 2019 The Authors

Published
2019

18. Outcomes and Treatment Strategies for Autoimmunity and Hyperinflammation in Patients with RAG Deficiency

Author

Farmer, J. R., Foldvari, Z., Ujhazi, B., De Ravin, S. S., Chen, K., Bleesing, J. J. H., Schuetz, C., Al-Herz, W., Abraham, R. S., Joshi, A. Y., Costa-Carvalho, B. T., Buchbinder, D., Booth, C., Reiff, A., Ferguson, P. J., Aghamohammadi, A., Abolhassani, H., Puck, J. M., Adeli, M., Cancrini, C., Palma, P., Bertaina, A., Locatelli, Franco, Di Matteo, G., Geha, R. S., Kanariou, M. G., Lycopoulou, L., Tzanoudaki, M., Sleasman, J. W., Parikh, S., Pinero, G., Fischer, B. M., Dbaibo, G., Unal, E., Patiroglu, T., Karakukcu, M., Al-Saad, K. K., Dilley, M. A., Pai, S. -Y., Dutmer, C. M., Gelfand, E. W., Geier, C. B., Eibl, M. M., Wolf, H. M., Henderson, L. A., Hazen, M. M., Bonfim, C., Wolska-Kusnierz, B., Butte, M. J., Hernandez, J. D., Nicholas, S. K., Stepensky, P., Chandrakasan, S., Miano, M., Westermann-Clark, E., Goda, V., Krivan, G., Holland, S. M., Fadugba, O., Henrickson, S. E., Ozen, A., Karakoc-Aydiner, E., Baris, S., Kiykim, A., Bredius, R., Hoeger, B., Boztug, K., Pashchenko, O., Neven, B., Moshous, D., de Villartay, J. -P., Bousfiha, A. A., Hill, H. R., Notarangelo, L. D., Walter, J. E., Locatelli F. (ORCID:0000-0002-7976-3654), Farmer, J. R., Foldvari, Z., Ujhazi, B., De Ravin, S. S., Chen, K., Bleesing, J. J. H., Schuetz, C., Al-Herz, W., Abraham, R. S., Joshi, A. Y., Costa-Carvalho, B. T., Buchbinder, D., Booth, C., Reiff, A., Ferguson, P. J., Aghamohammadi, A., Abolhassani, H., Puck, J. M., Adeli, M., Cancrini, C., Palma, P., Bertaina, A., Locatelli, Franco, Di Matteo, G., Geha, R. S., Kanariou, M. G., Lycopoulou, L., Tzanoudaki, M., Sleasman, J. W., Parikh, S., Pinero, G., Fischer, B. M., Dbaibo, G., Unal, E., Patiroglu, T., Karakukcu, M., Al-Saad, K. K., Dilley, M. A., Pai, S. -Y., Dutmer, C. M., Gelfand, E. W., Geier, C. B., Eibl, M. M., Wolf, H. M., Henderson, L. A., Hazen, M. M., Bonfim, C., Wolska-Kusnierz, B., Butte, M. J., Hernandez, J. D., Nicholas, S. K., Stepensky, P., Chandrakasan, S., Miano, M., Westermann-Clark, E., Goda, V., Krivan, G., Holland, S. M., Fadugba, O., Henrickson, S. E., Ozen, A., Karakoc-Aydiner, E., Baris, S., Kiykim, A., Bredius, R., Hoeger, B., Boztug, K., Pashchenko, O., Neven, B., Moshous, D., de Villartay, J. -P., Bousfiha, A. A., Hill, H. R., Notarangelo, L. D., Walter, J. E., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Background: Although autoimmunity and hyperinflammation secondary to recombination activating gene (RAG) deficiency have been associated with delayed diagnosis and even death, our current understanding is limited primarily to small case series. Objective: Understand the frequency, severity, and treatment responsiveness of autoimmunity and hyperinflammation in RAG deficiency. Methods: In reviewing the literature and our own database, we identified 85 patients with RAG deficiency, reported between 2001 and 2016, and compiled the largest case series to date of 63 patients with prominent autoimmune and/or hyperinflammatory pathology. Results: Diagnosis of RAG deficiency was delayed a median of 5 years from the first clinical signs of immune dysregulation. Most patients (55.6%) presented with more than 1 autoimmune or hyperinflammatory complication, with the most common etiologies being cytopenias (84.1%), granulomas (23.8%), and inflammatory skin disorders (19.0%). Infections, including live viral vaccinations, closely preceded the onset of autoimmunity in 28.6% of cases. Autoimmune cytopenias had early onset (median, 1.9, 2.1, and 2.6 years for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively) and were refractory to intravenous immunoglobulin, steroids, and rituximab in most cases (64.7%, 73.7%, and 71.4% for autoimmune hemolytic anemia, immune thrombocytopenia, and autoimmune neutropenia, respectively). Evans syndrome specifically was associated with lack of response to first-line therapy. Treatment-refractory autoimmunity/hyperinflammation prompted hematopoietic stem cell transplantation in 20 patients. Conclusions: Autoimmunity/hyperinflammation can be a presenting sign of RAG deficiency and should prompt further evaluation. Multilineage cytopenias are often refractory to immunosuppressive treatment and may require hematopoietic cell transplantation for definitive management.

Published
2019

19. Prevalence and clinical challenges among adults with primary immunodeficiency and recombination-activating gene deficiency

Author

Lawless, D, Geier, CB, Farmer, JR, Lango Allen, H, Thwaites, D, Atschekzei, F, Brown, M, Buchbinder, D, Burns, SO, Butte, MJ, Csomos, K, Deevi, SVV, Egner, W, Ehl, S, Eibl, MM, Fadugba, O, Foldvari, Z, Green, DM, Henrickson, SE, Holland, SM, John, T, Klemann, C, Kuijpers, TW, Moreira, F, Piller, A, Rayner-Matthews, P, Romberg, ND, Sargur, R, Schmidt, RE, Schröder, C, Schuetz, C, Sharapova, SO, Smith, KGC, Sogkas, G, Speckmann, C, Stirrups, K, Thrasher, AJ, Wolf, HM, Notarangelo, LD, Anwar, R, Boyes, J, Ujhazi, B, NIHR BioResource - Rare Diseases Consortium, Thaventhiran, J, Walter, JE, Savic, S, Lango Allen, Hana [0000-0002-7803-8688], Smith, Kenneth [0000-0003-3829-4326], Johnson, Kathleen [0000-0002-6823-3252], Thaventhiran, James [0000-0001-8616-074X], and Apollo - University of Cambridge Repository

Subjects
Adult, DNA-Binding Proteins, Homeodomain Proteins, Immunologic Deficiency Syndromes, Prevalence, Humans, Nuclear Proteins
Published
2018
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21. Impact of pre-transplant depression on outcomes of allogeneic and autologous hematopoietic stem cell transplantation

Author

El-Jawahri, A, Chen, Y-B, Brazauskas, R, He, N, Lee, SJ, Knight, JM, Majhail, N, Buchbinder, D, Schears, RM, Wirk, BM, Wood, WA, Ahmed, I, Aljurf, M, Szer, J, Beattie, SM, Battiwalla, M, Dandoy, C, Diaz, M-A, D'Souza, A, Freytes, CO, Gajewski, J, Gergis, U, Hashmi, SK, Jakubowski, A, Kamble, RT, Kindwall-Keller, T, Lazarus, HM, Malone, AK, Marks, DI, Meehan, K, Savani, BN, Olsson, RF, Rizzieri, D, Steinberg, A, Speckhart, D, Szwajcer, D, Schoemans, H, Seo, S, Ustun, C, Atsuta, Y, Dalal, J, Sales-Bonfim, C, Khera, N, Hahn, T, Saber, W, El-Jawahri, A, Chen, Y-B, Brazauskas, R, He, N, Lee, SJ, Knight, JM, Majhail, N, Buchbinder, D, Schears, RM, Wirk, BM, Wood, WA, Ahmed, I, Aljurf, M, Szer, J, Beattie, SM, Battiwalla, M, Dandoy, C, Diaz, M-A, D'Souza, A, Freytes, CO, Gajewski, J, Gergis, U, Hashmi, SK, Jakubowski, A, Kamble, RT, Kindwall-Keller, T, Lazarus, HM, Malone, AK, Marks, DI, Meehan, K, Savani, BN, Olsson, RF, Rizzieri, D, Steinberg, A, Speckhart, D, Szwajcer, D, Schoemans, H, Seo, S, Ustun, C, Atsuta, Y, Dalal, J, Sales-Bonfim, C, Khera, N, Hahn, T, and Saber, W

Published
2017

23. Selective laser melting of aluminum die-cast alloy

Author

Buchbinder, D., Meiners, W., Wissenbach, K., Poprawe, R., and Publica

Abstract

The additive production technology selective laser melting (SLM) is used for direct fabrication of metal-based functional components. SLM is one of the powder-bed based AM technologies. SLM is well established in serial production for dental restoration as well as for tooling. Main concern for industrial application remains the scope of processible materials and resulting mechanical properties. Toward processing of aluminum alloys commercially available systems exist with comparability in terms of applied process parameters and resulting mechanical properties remaining a challenge. Often no data are available concerning process parameters and mechanical properties. This holds especially for high-power SLM systems with increased build rates as a result of extended laser powers of up to 1 kW. Especially when processing aluminum alloys, the solidification conditions significantly affect the resulting microstructure in terms of size of dendrites and grains. Consequently, the present paper systematically investigates and correlates the process parameters (e.g., laser power, scan speed, layer orientation, preheating, etc.) on the microstructure (electron backscatter diffraction and texture) and resultant mechanical properties (hardness, tensile strength, yield strength, and breaking elongation) for aluminum die-cast alloys. At this, underlying phenomena for typically observed anisotropy of mechanical properties in dependence on layer orientation are further specified.

Published
2015

25. Additive manufactured AlSi10Mg samples using Selective Laser Melting (SLM)

Author

Brandl, E., Heckenberger, U., Holzinger, V., Buchbinder, D., and Publica

Abstract

In order to produce serial parts via additive layer manufacturing, the fatigue performance can be a critical attribute. In this paper, the microstructure, high cycle fatigue (HCF), and fracture behavior of additive manufactured AlSi10Mg samples are investigated. The samples were manufactured by a particular powder-bed process called Selective Laser Melting (SLM) and machined afterwards. 91 samples were manufactured without (30°C) and with heating (300°C) of the building platform and in different directions (0°, 45°, 90°). Samples were tested in the peak-hardened (T6) and as-built condition. The Wöhler curves were interpolated by a Weibull distribution. The results were analysed statistically by design of experiments, correlation analysis, and marginal means plots. The investigations show that the post heat treatment has the most considerable effect and the building direction has the least considerable effect on the fatigue resistance. The fatigue resistance of the sampl es, however, is high in comparison to the standard DIN EN 1706. The combination of 300°C platform heating and peak-hardening is a valuable approach to increase the fatigue resistance and neutralize the differences in fatigue life for the 0°, 45°, and 90° directions.

Published
2012

26. Mit Selective Laser Melting auf dem Weg zur Serienproduktion?

Author

Bremen, S., Buchbinder, D., Meiners, W., Wissenbach, K., and Publica

Abstract

Die fortschreitende Globalisierung und die damit einhergehende Sättigung und Segmentierung der Märkte erhöht die Variantenzahl produzierender Unternehmen und verstärkt die Nachfrage an individualisierten Produkten. Um in diesem Wettbewerb bestehen zu können, setzen Unternehmen in Hochlohnländern auf eine Kombination aus individuellen und gleichzeitig qualitativ hochwertigen Produkten. Die Herausforderung besteht in diesem Zusammenhang darin, möglichst schnell auf individuelle Kundenwünsche zu reagieren und gleichzeitig die Kosten niedrig zu halten. Ein vielversprechender Ansatz dieses Problem zu lösen bietet die Technologie der generativen Fertigung, die in den letzten Jahren ihr technologisches Potenzial erheblich weiterentwickelt hat.

Published
2011

27. Untersuchung zur Reduzierung des Verzugs durch Vorwärmung bei der Herstellung von Aluminiumbauteilen mittels SLM

Author

Buchbinder, D., Schilling, G., Meiners, W., Pirch, N., Wissenbach, K., and Publica

Subjects
Schrumpfung, Aluminium, Spannungen, Riß, SLM, Verzug
Abstract

Das generative Fertigungsverfahren Selective Laser Melting (SLM) wird zur direkten Herstellung von metallischen Funktionsbauteilen verwendet. Während des Bauprozesses entstehen durch den schichtweisen Aufbau und die lokale Energieeinbringung mittels eines fokussierten Laserstrahls thermisch induzierte Eigenspannungen, die zu Verzug des Bauteils oder von Bauteilbereichen führen können. Üblicherweise werden die Verzüge durch Stützstrukturen zwischen Bauteil und Substratplatte verhindert. Jedoch ist es nicht immer möglich alle Bereiche eines Bauteils, je nach Komplexität der Geometrie oder Zugänglichkeit, mit Stützstrukturen zu versehen bzw. diese wieder zu entfernen. Durch eine Vorwärmung der Substratplatte während des Bauprozesses können die Verzüge reduziert oder ganz vermieden werden. Jedoch ist bisher keine systematische Untersuchung des Einflusses der Vorwärmung auf Verzüge von Aluminium Bauteilen durchgeführt worden. Ziel dieser Arbeiten ist daher die systematische Untersuchung der Auswirkung einer Vorwärmung beim SLM von Aluminiumbauteilen und die Ermittlung der geeigneten Vorwärmtemperatur, bei der nahezu keine Verzüge mehr entstehen. Eine signifikante Verzugsreduzierung im Vergleich zu den Verzügen ohne Vorwärmung zeigt sich ab einer Vorwärmtemperatur von 150°C. Bei einer Vorwärmtemperatur von 250°C sind im Rahmen der Messgenauigkeit unabhängig von der untersuchten Twincantilever Testgeometrie keine Verzüge mehr feststellbar. Neben der Reduzierung der Verzüge verhindert die Vorwärmung außerdem spannungsbedingte Risse im Bauteil, die ohne Vorwärmung zum Abreißen von Teilen der Testgeometrie führen können. Mit 90 HV 0,1 bei 250°C Vorwärmtemperatur ist die Härte größer als die geforderte Mindesthärte nach DIN EN 1706 von Druckgussbauteilen aus dem Werkstoff AlSi10Mg. Aus diesem Ergebnis kann abgeleitet werden, dass eine Vorwärmtemperatur von 250°C geeignet ist, Bauteile aus dem Werkstoff AlSi10Mg mit SLM defektfrei und prozesssicher herzustellen und Verzüge vollständig zu vermeiden.

Published
2011

35. Second malignancies after autologous hematopoietic cell transplantation in children

Author

Danner-Koptik, K E, primary, Majhail, N S, additional, Brazauskas, R, additional, Wang, Z, additional, Buchbinder, D, additional, Cahn, J-Y, additional, Dilley, K J, additional, Frangoul, H A, additional, Gross, T G, additional, Hale, G A, additional, Hayashi, R J, additional, Hijiya, N, additional, Kamble, R T, additional, Lazarus, H M, additional, Marks, D I, additional, Reddy, V, additional, Savani, B N, additional, Warwick, A B, additional, Wingard, J R, additional, Wood, W A, additional, Sorror, M L, additional, and Jacobsohn, D A, additional

Published
2012
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36. Late Effects in Hematopoietic Cell Transplant Recipients with Acquired Severe Aplastic Anemia: A Report from the Late Effects Working Committee of the Center for International Blood and Marrow Transplant Research (CIBMTR)

Author

Buchbinder, D., primary, Nugent, D., additional, Brazauskas, R., additional, Wang, Z., additional, Aljurf, M., additional, Cairo, M.S., additional, Chow, R., additional, Duncan, C., additional, Eldjerou, L.K., additional, Gupta, V., additional, Hale, G.A., additional, Halter, J., additional, Hayes-Lattin, B.M., additional, Hsu, J.W., additional, Jacobsohn, D.A., additional, Kamble, R.T., additional, Kasow, K.A., additional, Lazarus, H.M., additional, Mehta, P., additional, Myers, K.C., additional, Parsons, S.K., additional, Passweg, J.R., additional, Pidala, J., additional, Reddy, V., additional, Sales-Bonfim, C.M., additional, Savani, B.N., additional, Seber, A., additional, Sorror, M.L., additional, Steinberg, A., additional, Wood, W.A., additional, Wall, D.A., additional, Winiarski, J.H., additional, Yu, L.C., additional, and Majhail, N.S., additional

Published
2012
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