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102. Venetoclax and Navitoclax in Combination with Chemotherapy in Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma

Author

Pullarkat, Vinod A., primary, Lacayo, Norman J., additional, Jabbour, Elias, additional, Rubnitz, Jeffrey E., additional, Bajel, Ashish, additional, Laetsch, Theodore W., additional, Leonard, Jessica, additional, Colace, Susan I., additional, Khaw, Seong Lin, additional, Fleming, Shaun A., additional, Mattison, Ryan J., additional, Norris, Robin, additional, Opferman, Joseph T., additional, Roberts, Kathryn G., additional, Zhao, Yaqi, additional, Qu, Chunxu, additional, Badawi, Mohamed, additional, Schmidt, Michelle, additional, Tong, Bo, additional, Pesko, John C., additional, Sun, Yan, additional, Ross, Jeremy A., additional, Vishwamitra, Deeksha, additional, Rosenwinkel, Lindsey, additional, Kim, Su Young, additional, Jacobson, Amanda, additional, Mullighan, Charles G., additional, Alexander, Thomas B., additional, and Stock, Wendy, additional

Published
2021
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103. The role of 18F‐Fluorodeoxyglucose Positron Emission Tomography/Computed Tomography (FDG PET/CT) in assessment of complex invasive fungal disease and opportunistic co‐infections in patients with acute leukemia prior to allogeneic hematopoietic cell transplant

Author

Longhitano, Anthony, primary, Alipour, Ramin, additional, Khot, Amit, additional, Bajel, Ashish, additional, Antippa, Phillip, additional, Slavin, Monica, additional, and Thursky, Karin, additional

Published
2020
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105. A Prospective Haploidentical Peripheral Blood Stem Cell Transplant Study Using a Pre-Defined Conditioning Regimen Intensity Based on Age and the Hematopoietic Cell Transplantation Comorbidity Index- Anzhit 1: Encouraging Preliminary Survival Outcomes at One Year Follow up

Author

Moore, John, primary, Kwan, John, additional, Gottlieb, David, additional, Bajel, Ashish, additional, Ritchie, David, additional, Yeung, David T, additional, Greenwood, Matthew, additional, Purtill, Duncan, additional, Tran, Steven, additional, Aarons, Donna, additional, and Hamad, Nada, additional

Published
2020
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108. Bone Marrow Transplant Society of Australia and New Zealand COVID‐19 consensus position statement

Author

Hamad, Nada, primary, Gottlieb, David, additional, Ritchie, David, additional, Kennedy, Glen, additional, Watson, Anne M., additional, Greenwood, Matthew, additional, Doocey, Richard, additional, Perera, Travis, additional, Spencer, Andrew, additional, Wong, Eric, additional, O'Brien, Tracey, additional, Shaw, Peter, additional, Conyers, Rachel, additional, Cole, Theresa, additional, Milliken, Sam, additional, Bardy, Peter, additional, Larsen, Stephen, additional, Lai, Hock, additional, Butler, Andrew, additional, Fraser, Chris, additional, Bajel, Ashish, additional, Butler, Jason, additional, Kerridge, Ian, additional, and Purtill, Duncan, additional

Published
2020
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116. Effect of donor age on adult unrelated donor haemopoietic cell transplant outcome: the Australian experience.

Author

Nivison‐Smith, Ian, Bajel, Ashish, Dodds, Anthony J., Gottlieb, David, Hamad, Nada, Kennedy, Glen, Kerridge, Ian, Ma, David D. F., Milliken, Samuel, Moore, John, Purtill, Duncan, and Szer, Jeff

Subjects
*AGE distribution, *HEMATOPOIETIC stem cell transplantation, *ORGAN donors
Abstract

Background: Results have been varied regarding the effect of donor age on the outcome of unrelated donor haemopoietic cell transplantation (HCT). Aims: To determine the influence of donor age on adult unrelated donor HCT outcome in Australia. Methods: Patients were included in the study if they were aged 16 years or above and underwent first allogeneic unrelated donor HCT in Australia for the indications of acute lymphoblastic leukaemia (ALL), acute myelogenous leukaemia (AML), chronic myelogenous leukaemia (CML) or myelodysplastic syndromes (MDS) between the years of 2001 and 2014 inclusive. The main outcome measure was overall survival (OS), which was tested against independent variables using univariate Kaplan–Meier methods and multivariate Cox regression. Results: A total of 1158 unrelated donor HCT were represented in the data. Cumulative incidences of engraftment, transplant related mortality (TRM), acute graft‐versus‐host disease (GvHD), chronic GvHD and relapse were not significantly affected by donor age. OS probability at 5 years post‐transplant was 48.3%. In multivariate analysis of OS, year of transplant 2001–2007, recipient age 40 years or greater, poor risk disease, human leukocyte antigen (HLA) match less than 6/6 and poor performance status at transplant (Karnofsky scale) were independently significant adverse OS risk factors. Donor age was not a significant risk factor for OS in univariate or multivariate analysis. Conclusions: The conclusion from this study was that donor age (up to 59 years) did not influence post‐transplant outcome among adult unrelated donor HCT performed in Australia for haematologic malignancies. [ABSTRACT FROM AUTHOR]

Published
2022
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117. Consensus guidelines for the diagnosis and management of invasive aspergillosis, 2021.

Author

Douglas, Abby P., Smibert, Olivia. C., Bajel, Ashish, Halliday, Catriona L., Lavee, Orly, McMullan, Brendan, Yong, Michelle K., van Hal, Sebastiaan J., and Chen, Sharon C.‐A.

Subjects
ASPERGILLOSIS diagnosis, ASPERGILLOSIS treatment, BIOMARKERS, ANTIMICROBIAL stewardship, ANTIFUNGAL agents, BIOPSY, ANTI-infective agents, MEDICAL protocols, ASPERGILLOSIS, ENDOPHTHALMITIS, OSTEOMYELITIS, KERATITIS, DISEASE complications
Abstract

Invasive aspergillosis (IA) in haematology/oncology patients presents as primary infection or breakthrough infection, which can become refractory to antifungal treatment and has a high associated mortality. Other emerging patient risk groups include patients in the intensive care setting with severe respiratory viral infections, including COVID‐19. These guidelines present key diagnostic and treatment recommendations in light of advances in knowledge since the previous guidelines in 2014. Culture and histological‐based methods remain central to the diagnosis of IA. There is increasing evidence for the utility of non‐culture methods employing fungal biomarkers in pre‐emptive screening for infection, as well as for IA diagnosis when used in combination. Although azole resistance appears to be uncommon in Australia, susceptibility testing of clinical Aspergillus fumigatus complex isolates is recommended. Voriconazole remains the preferred first‐line antifungal agent for treating primary IA, including for extrapulmonary disease. Recommendations for paediatric treatment broadly follow those for adults. For breakthrough and refractory IA, a change in class of antifungal agent is strongly recommended, and agents under clinical trial may need to be considered. Newer immunological‐based imaging modalities warrant further study, while surveillance for IA and antifungal resistance remain essential to informing the relevance of current treatment recommendations. [ABSTRACT FROM AUTHOR]

Published
2021
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118. Intravenous Busulfan Compared with Total Body Irradiation Pretransplant Conditioning for Adults with Acute Lymphoblastic Leukemia

Author

Kebriaei, Partow, Anasetti, Claudio, Zhang, Mei Jie, Wang, Hai Lin, Aldoss, Ibrahim, de Lima, Marcos, Khoury, H. Jean, Sandmaier, Brenda M., Horowitz, Mary M., Artz, Andrew, Bejanyan, Nelli, Ciurea, Stefan, Lazarus, Hillard M., Gale, Robert Peter, Litzow, Mark, Bredeson, Christopher, Seftel, Matthew D., Pulsipher, Michael A., Boelens, Jaap Jan, Alvarnas, Joseph, Champlin, Richard, Forman, Stephen, Pullarkat, Vinod, Weisdorf, Daniel, Marks, David I., Hogan, William, Battiwalla, Minoo, Copelan, Edward, Hildebrandt, Gerhard, Ganguly, Sid, Majhail, Navneet, Woolfrey, Ann, Nivison-Smith, Ian, Hertzberg, Mark, Diaz, Miguel Angel, Jakubowski, Ann, Ustun, Celalettin, Yong, Agnes, Freytes, Cesar, DeFilipp, Zachariah, Inamoto, Yoshi, Cahn, Jean Yves, Savani, Bipin, Yared, Jean, Bajel, Ashish, Bacher, Ulrike, Uy, Geoffrey, Rizzieri, David, Wieduwilt, Matthew, Bierings, Marc, and Acute Leukemia Committee of the CIBMTR

Subjects
Transplantation, Total body irradiation, Hematology, Acute lymphoblastic leukemia, Busulfan, Allogeneic transplant
Abstract

Total body irradiation (TBI) has been included in standard conditioning for acute lymphoblastic leukemia (ALL) before hematopoietic cell transplantation (HCT). Non-TBI regimens have incorporated busulfan (Bu) to decrease toxicity. This retrospective study analyzed TBI and Bu on outcomes of ALL patients 18–60 years old, in first or second complete remission (CR), undergoing HLA-compatible sibling, related, or unrelated donor HCT, who reported to the Center for International Blood and Marrow Transplant Research from 2005 to 2014. TBI plus etoposide (25%) or cyclophosphamide (75%) was used in 819 patients, and intravenous Bu plus fludarabine (41%), clofarabine (30%), cyclophosphamide (15%), or melphalan (13%) was used in 299 patients. Bu-containing regimens were analyzed together, since no significant differences for patient outcomes were noted between them. Bu patients were older, with better performance status; took longer to achieve first CR and receive HCT; were treated more recently; and were more likely to receive peripheral blood grafts, antithymocyte globulin, or tyrosine kinase inhibitors. With median follow-up of 3.6 years for Bu and 5.3 years for TBI, adjusted 3-year outcomes showed treatment-related mortality Bu 19% versus TBI 25% (P =.04); relapse Bu 37% versus TBI 28% (P =.007); disease-free survival (DFS) Bu 45% versus TBI 48% (P =.35); and overall survival (OS) Bu 57% versus TBI 53% (P =.35). In multivariate analysis, Bu patients had higher risk of relapse (relative risk, 1.46; 95% confidence interval, 1.15 to 1.85; P =.002) compared with TBI patients. Despite the higher relapse, Bu-containing conditioning led to similar OS and DFS following HCT for ALL.

Published
2018

120. Preliminary Minimal Residual Disease Analysis of the Australasian Leukaemia & Lymphoma Group (ALLG) ALL8 Study of Front-Line Blinatumomab with Chemotherapy in Adults with Ph Negative B-Cell Acute Lymphoblastic Leukaemia

Author

Fleming, Shaun, primary, Venn, Nicola, additional, Reynolds, John, additional, Nguyen, Uyen, additional, Kwan, John, additional, Moore, John, additional, Yeung, David T, additional, Leahy, Michael F., additional, Greenwood, Matthew, additional, Verner, Emma, additional, Bajel, Ashish, additional, Berkahn, Leanne, additional, Wei, Andrew H., additional, and Sutton, Rosemary, additional

Published
2019
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121. Safety and Efficacy of Venetoclax in Combination with Navitoclax in Adult and Pediatric Relapsed/Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma

Author

Lacayo, Norman J., primary, Pullarkat, Vinod A., primary, Stock, Wendy, primary, Jabbour, Elias, primary, Bajel, Ashish, primary, Rubnitz, Jeffrey, primary, Leonard, Jessica, primary, Mullighan, Charles G., primary, Khaw, Seong Lin, primary, Vear, Susan I., primary, Opferman, Joseph T., primary, Salem, Ahmed Hamed, primary, Schmidt, Michelle, primary, Tong, Bo, primary, Zhou, Lang, primary, Ross, Jeremy A., primary, Bensman, Lindsey, primary, Jacobson, Amanda, primary, Norris, Robin, primary, and Alexander, Thomas, primary

Published
2019
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123. A Phase 1b Study Evaluating the Safety and Efficacy of Venetoclax As Monotherapy or in Combination with Azacitidine for the Treatment of Relapsed/Refractory Myelodysplastic Syndrome

Author

Zeidan, Amer M., primary, Pollyea, Daniel A, primary, Garcia, Jacqueline S, primary, Brunner, Andrew, primary, Roncolato, Fernando, primary, Borate, Uma, primary, Odenike, Olatoyosi, primary, Bajel, Ashish R., primary, Watson, Anne Marie, primary, Götze, Katharina, primary, Nolte, Florian, primary, Tan, Peter T., primary, Hong, Wan-Jen, primary, Dunbar, Martin, primary, Zhou, Ying, primary, Gressick, Lori, primary, Ainsworth, William, primary, Harb, Jason, primary, Salem, Ahmed Hamed, primary, Hayslip, John, primary, Swords, Ronan, primary, and Garcia-Manero, Guillermo, primary

Published
2019
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125. Combination BCL-2 Inhibitor Therapy with Venetoclax and Navitoclax in Patients with Relapsed/Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma

Author

Jabbour, Elias, primary, Mullighan, Charles G., additional, Pullarkat, Vinod A., additional, Lacayo, Norman J., additional, Bajel, Ashish, additional, Rubnitz, Jeffrey, additional, Leonard, Jessica, additional, Khaw, Seong Lin, additional, Vear, Susan I., additional, Alexander, Thomas, additional, Norris, Robin, additional, Salem, Ahmed, additional, Schmidt, Michelle, additional, Tong, Bo, additional, Zhou, Ying, additional, Ross, Jeremy A., additional, Bensman, Lindsey, additional, Jacobson, Amanda, additional, and Stock, Wendy, additional

Published
2019
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129. The role of 18F‐Fluorodeoxyglucose Positron Emission Tomography/Computed Tomography (FDG PET/CT) in assessment of complex invasive fungal disease and opportunistic co‐infections in patients with acute leukemia prior to allogeneic hematopoietic cell transplant

Author

Longhitano, Anthony, Alipour, Ramin, Khot, Amit, Bajel, Ashish, Antippa, Phillip, Slavin, Monica, and Thursky, Karin

Subjects
POSITRON emission tomography computed tomography, MYCOSES, COMPUTED tomography, ACUTE leukemia, MIXED infections, AIDS-related opportunistic infections, PULMONARY aspergillosis
Abstract

Introduction: Individuals diagnosed with acute lymphoid and myeloid malignancies are at significant risk of invasive fungal and bacterial infections secondary to their marked immunocompromised states with a significant high risk of mortality. The role of metabolic imaging with 18F‐Fluorodeoxyglucose (FDG) Positron Emission Tomography/Computed Tomography (PET/CT) has been increasingly recognized in optimizing the diagnosis of invasive infection, monitoring the response to therapy and guiding the duration of antimicrobial therapy or need to escalate to surgical intervention. Methods: Two distinct cases of pulmonary co‐infection of rare fungal and bacterial pathogens are explored in severely immunocompromised individuals where FDG PET/CT aided both patients to make a full recovery and transition to HCT. The first case explores mixed Scedosporium apiospermum and Rhizomucor pulmonary infection on a background of T cell/myeloid mixed phenotype acute leukemia ultimately warranting long‐term antifungal therapy and lobectomy prior to HCT. The second case explores Fusarium and Nocardia pulmonary infection on a background of relapsed AML also warranting surgical resection with lobectomy and long‐term antimicrobials prior to transition to HCT. Discussion: The cases highlight the utility of FDG PET/CT to support the diagnosis of infections, including the presence or absence of disseminated infection, and to provide highly sensitive monitoring of the infection over time. FDG PET/CT played a key role in directing therapy duration decisions and prompted the necessity for surgical intervention. Ultimately, the use of FDG PET/CT allowed for a successful transition to HCT highlighting its value in this clinical setting. Conclusion: FDG PET/CT has an emerging role in the diagnostic and monitoring pathway for complex infections in high‐risk immunocompromised patients. [ABSTRACT FROM AUTHOR]

Published
2021
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133. Preliminary Results of a Phase 2a Dose Optimization Study of ASLAN003 (DHODH inhibitor) in Acute Myeloid Leukemia (AML) Patients Who Are Ineligible for Standard Therapy; Early Signs of Activity

Author

Ting, Stephen B, primary, Ng, Chin Hin, additional, Bajel, Ashish, additional, Hwang, William YK, additional, Ho, Shir-Jing, additional, Chng, Wee-Joo, additional, McHale, Mark, additional, Hsieh, Chih-Yi, additional, Shih, Hsuan-Jen, additional, McIntyre, Nicola, additional, Kwek, Jamie, additional, Chang, Wei-Ling, additional, and Lindmark, Bertil, additional

Published
2018
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134. Venetoclax and Navitoclax in Patients with Relapsed or Refractory Acute Lymphoblastic Leukemia and Lymphoblastic Lymphoma

Author

Alexander, Thomas, primary, Lacayo, Norman J., additional, Pullarkat, Vinod A., additional, Jabbour, Elias J., additional, Khaw, Seong Lin, additional, Mullighan, Charles G., additional, Bajel, Ashish, additional, Leonard, Jessica, additional, Norris, Robin, additional, Rubnitz, Jeffrey, additional, Vear, Susan I., additional, Schmidt, Michelle, additional, Tong, Bo, additional, Zhou, Ying, additional, Ross, Jeremy A., additional, Rosenwinkel, Lindsey, additional, Jacobson, Amanda, additional, Kim, Su Young, additional, and Stock, Wendy, additional

Published
2018
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135. Abstract 1366:MBD4guards against DNA damage from methylcytosine deamination

Author

Chew, Edward, primary, Sanders, Mathijs A., additional, Flensburg, Christoffer, additional, Zeilemaker, Annelieke, additional, Miller, Sarah E., additional, Hinai, Adil S. al, additional, Bajel, Ashish, additional, Luiken, Bram, additional, Rijken, Melissa, additional, Mclennan, Tamara, additional, Hoogenboezem, Remco M., additional, Kavelaars, François G., additional, Blewitt, Marnie E., additional, Bindels, Eric M., additional, Alexander, Warren S., additional, Löwenberg, Bob, additional, Roberts, Andrew W., additional, Majewski, Ian J., additional, and Valk, Peter J., additional

Published
2018
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136. Intravenous Busulfan Compared with Total Body Irradiation Pretransplant Conditioning for Adults with Acute Lymphoblastic Leukemia

Author

Kebriaei, Partow, primary, Anasetti, Claudio, additional, Zhang, Mei-Jie, additional, Wang, Hai-Lin, additional, Aldoss, Ibrahim, additional, de Lima, Marcos, additional, Khoury, H. Jean, additional, Sandmaier, Brenda M., additional, Horowitz, Mary M., additional, Artz, Andrew, additional, Bejanyan, Nelli, additional, Ciurea, Stefan, additional, Lazarus, Hillard M., additional, Gale, Robert Peter, additional, Litzow, Mark, additional, Bredeson, Christopher, additional, Seftel, Matthew D., additional, Pulsipher, Michael A., additional, Boelens, Jaap-Jan, additional, Alvarnas, Joseph, additional, Champlin, Richard, additional, Forman, Stephen, additional, Pullarkat, Vinod, additional, Weisdorf, Daniel, additional, Marks, David I., additional, Hogan, William, additional, Battiwalla, Minoo, additional, Copelan, Edward, additional, Hildebrandt, Gerhard, additional, Ganguly, Sid, additional, Majhail, Navneet, additional, Woolfrey, Ann, additional, Nivison-Smith, Ian, additional, Hertzberg, Mark, additional, Diaz, Miguel Angel, additional, Jakubowski, Ann, additional, Ustun, Celalettin, additional, Yong, Agnes, additional, Freytes, Cesar, additional, DeFilipp, Zachariah, additional, Inamoto, Yoshi, additional, Cahn, Jean-Yves, additional, Savani, Bipin, additional, Yared, Jean, additional, Bajel, Ashish, additional, Bacher, Ulrike, additional, Uy, Geoffrey, additional, Rizzieri, David, additional, Wieduwilt, Matthew, additional, Schultz, Kirk, additional, Grunwald, Michael, additional, Kamble, Rammurti, additional, Qayed, Muna, additional, Brammer, Jonathan, additional, Ballen, Karen, additional, Khera, Nandita, additional, Schouten, Harry, additional, Bierings, Marc, additional, Kanakry, Christopher, additional, Wood, William Allen, additional, Saad, Ayman, additional, Savoie, Lynn, additional, Hamilton, Betty Ky, additional, Aljurf, Mahmoud, additional, Vij, Ravi, additional, Pawarode, Attaphol, additional, Olsson, Richard, additional, Wirk, B. Mona, additional, Beitinjaneh, Amer, additional, Seo, Sachiko, additional, Cerny, Jan, additional, Akpek, Gorgun, additional, Norkin, Maxim, additional, Nishihori, Taiga, additional, Lazaryan, Aleksandr, additional, and Sabloff, Mitchell, additional

Published
2018
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137. Intravenous Busulfan Compared with Total Body Irradiation Pretransplant Conditioning for Adults with Acute Lymphoblastic Leukemia

Author

CTI Nierkens, Child Health, Regenerative Medicine and Stem Cells, Haematologie, Kebriaei, Partow, Anasetti, Claudio, Zhang, Mei Jie, Wang, Hai Lin, Aldoss, Ibrahim, de Lima, Marcos, Khoury, H. Jean, Sandmaier, Brenda M., Horowitz, Mary M., Artz, Andrew, Bejanyan, Nelli, Ciurea, Stefan, Lazarus, Hillard M., Gale, Robert Peter, Litzow, Mark, Bredeson, Christopher, Seftel, Matthew D., Pulsipher, Michael A., Boelens, Jaap Jan, Alvarnas, Joseph, Champlin, Richard, Forman, Stephen, Pullarkat, Vinod, Weisdorf, Daniel, Marks, David I., Hogan, William, Battiwalla, Minoo, Copelan, Edward, Hildebrandt, Gerhard, Ganguly, Sid, Majhail, Navneet, Woolfrey, Ann, Nivison-Smith, Ian, Hertzberg, Mark, Diaz, Miguel Angel, Jakubowski, Ann, Ustun, Celalettin, Yong, Agnes, Freytes, Cesar, DeFilipp, Zachariah, Inamoto, Yoshi, Cahn, Jean Yves, Savani, Bipin, Yared, Jean, Bajel, Ashish, Bacher, Ulrike, Uy, Geoffrey, Rizzieri, David, Wieduwilt, Matthew, Bierings, Marc, Acute Leukemia Committee of the CIBMTR, CTI Nierkens, Child Health, Regenerative Medicine and Stem Cells, Haematologie, Kebriaei, Partow, Anasetti, Claudio, Zhang, Mei Jie, Wang, Hai Lin, Aldoss, Ibrahim, de Lima, Marcos, Khoury, H. Jean, Sandmaier, Brenda M., Horowitz, Mary M., Artz, Andrew, Bejanyan, Nelli, Ciurea, Stefan, Lazarus, Hillard M., Gale, Robert Peter, Litzow, Mark, Bredeson, Christopher, Seftel, Matthew D., Pulsipher, Michael A., Boelens, Jaap Jan, Alvarnas, Joseph, Champlin, Richard, Forman, Stephen, Pullarkat, Vinod, Weisdorf, Daniel, Marks, David I., Hogan, William, Battiwalla, Minoo, Copelan, Edward, Hildebrandt, Gerhard, Ganguly, Sid, Majhail, Navneet, Woolfrey, Ann, Nivison-Smith, Ian, Hertzberg, Mark, Diaz, Miguel Angel, Jakubowski, Ann, Ustun, Celalettin, Yong, Agnes, Freytes, Cesar, DeFilipp, Zachariah, Inamoto, Yoshi, Cahn, Jean Yves, Savani, Bipin, Yared, Jean, Bajel, Ashish, Bacher, Ulrike, Uy, Geoffrey, Rizzieri, David, Wieduwilt, Matthew, Bierings, Marc, and Acute Leukemia Committee of the CIBMTR

Published
2018

138. Intact TP-53 function is essential for sustaining durable responses to BH3-mimetic drugs in leukemias

Author

Thijssen, Rachel, Diepstraten, Sarah T., Moujalled, Donia, Chew, Edward, Flensburg, Christoffer, Shi, Melissa X., Dengler, Michael A., Litalien, Veronique, MacRaild, Sarah, Chen, Maoshan, Anstee, Natasha S., Reljić, Boris, Gabriel, Sarah S., Djajawi, Tirta M., Riffkin, Chris D., Aubrey, Brandon J., Chang, Catherine, Tai, Lin, Xu, Zhen, Morley, Thomas, Pomilio, Giovanna, Bruedigam, Claudia, Kallies, Axel, Stroud, David A., Bajel, Ashish, Kluck, Ruth M., Lane, Steven W., Schoumacher, Marie, Banquet, Sébastien, Majewski, Ian J., Strasser, Andreas, Roberts, Andrew W., Huang, David C.S., Brown, Fiona C., Kelly, Gemma L., and Wei, Andrew H.

Abstract

Selective targeting of BCL-2 with the BH3-mimetic venetoclax has been a transformative treatment for patients with various leukemias. TP-53 controls apoptosis upstream of where BCL-2 and its prosurvival relatives, such as MCL-1, act. Therefore, targeting these prosurvival proteins could trigger apoptosis across diverse blood cancers, irrespective of TP53mutation status. Indeed, targeting BCL-2 has produced clinically relevant responses in blood cancers with aberrant TP-53. However, in our study, TP53-mutated or -deficient myeloid and lymphoid leukemias outcompeted isogenic controls with intact TP-53, unless sufficient concentrations of BH3-mimetics targeting BCL-2 or MCL-1 were applied. Strikingly, tumor cells with TP-53 dysfunction escaped and thrived over time if inhibition of BCL-2 or MCL-1 was sublethal, in part because of an increased threshold for BAX/BAK activation in these cells. Our study revealed the key role of TP-53 in shaping long-term responses to BH3-mimetic drugs and reconciled the disparate pattern of initial clinical response to venetoclax, followed by subsequent treatment failure among patients with TP53-mutant chronic lymphocytic leukemia or acute myeloid leukemia. In contrast to BH3-mimetics targeting just BCL-2 or MCL-1 at doses that are individually sublethal, a combined BH3-mimetic approach targeting both prosurvival proteins enhanced lethality and durably suppressed the leukemia burden, regardless of TP53mutation status. Our findings highlight the importance of using sufficiently lethal treatment strategies to maximize outcomes of patients with TP53-mutant disease. In addition, our findings caution against use of sublethal BH3-mimetic drug regimens that may enhance the risk of disease progression driven by emergent TP53-mutant clones.

Published
2021
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139. Diagnosis of rare subtypes of acute myeloid leukaemia and related neoplasms

Author

George, Tracy I. and Bajel, Ashish

Abstract

The diagnosis of acute myeloid leukaemia and related neoplasms in adults is challenging as this requires the integration of clinical findings, morphology, immunophenotype, cytogenetics, and molecular genetic findings. Lack of familiarity with rare subtypes of acute leukaemia hinders the diagnosis. In this review, we will describe diagnostic findings of several rare acute myeloid leukaemias and related neoplasms that primarily occur in adults including information on presentation, morphology, immunophenotype, genetics, differential diagnosis, and prognosis. Leukaemias discussed include blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukaemia with t(6;9) (p23;q34.1); DEK-NUP214, acute myeloid leukaemia with inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2); GATA2, MECOM, acute myeloid leukaemia with BCR-ABL1, acute leukaemias of ambiguous lineage, acute myeloid leukaemia with mutated RUNX1, pure erythroid leukaemia, acute panmyelosis with myelofibrosis, and acute basophilic leukaemia. Case studies with morphological features of the nine subtypes of acute myeloid leukaemia and related neoplasms have been included, and additional evidence available since publication of the 2016 World Health Organization Classification has been added to each subtype.

Published
2021
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140. Germline loss of MBD4 predisposes to leukaemia due to a mutagenic cascade driven by 5mC

Author

Sanders, Mathijs A., primary, Chew, Edward, additional, Flensburg, Christoffer, additional, Zeilemaker, Annelieke, additional, Miller, Sarah E., additional, al Hinai, Adil S., additional, Bajel, Ashish, additional, Luiken, Bram, additional, Rijken, Melissa, additional, Mclennan, Tamara, additional, Hoogenboezem, Remco M., additional, Kavelaars, François G., additional, Blewitt, Marnie E., additional, Bindels, Eric M., additional, Alexander, Warren S., additional, Löwenberg, Bob, additional, Roberts, Andrew W., additional, Valk, Peter J.M., additional, and Majewski, Ian J., additional

Published
2017
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141. A phase 2 study of momelotinib, a potent JAK1 and JAK2 inhibitor, in patients with polycythemia vera or essential thrombocythemia

Author

Verstovsek, Srdan, primary, Courby, Stephane, additional, Griesshammer, Martin, additional, Mesa, Ruben A., additional, Brachmann, Carrie Baker, additional, Kawashima, Jun, additional, Maltzman, Julia D., additional, Shao, Lixin, additional, Xin, Yan, additional, Huang, Daniel, additional, and Bajel, Ashish, additional

Published
2017
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142. Increasing use of allogeneic hematopoietic cell transplantation in patients aged 70 years and older in the United States

Author

Muffly, Lori, primary, Pasquini, Marcelo C., additional, Martens, Michael, additional, Brazauskas, Ruta, additional, Zhu, Xiaochun, additional, Adekola, Kehinde, additional, Aljurf, Mahmoud, additional, Ballen, Karen K., additional, Bajel, Ashish, additional, Baron, Frederic, additional, Battiwalla, Minoo, additional, Beitinjaneh, Amer, additional, Cahn, Jean-Yves, additional, Carabasi, Mathew, additional, Chen, Yi-Bin, additional, Chhabra, Saurabh, additional, Ciurea, Stefan, additional, Copelan, Edward, additional, D’Souza, Anita, additional, Edwards, John, additional, Foran, James, additional, Freytes, Cesar O., additional, Fung, Henry C., additional, Gale, Robert Peter, additional, Giralt, Sergio, additional, Hashmi, Shahrukh K., additional, Hildebrandt, Gerhard C., additional, Ho, Vincent, additional, Jakubowski, Ann, additional, Lazarus, Hillard, additional, Luskin, Marlise R., additional, Martino, Rodrigo, additional, Maziarz, Richard, additional, McCarthy, Philip, additional, Nishihori, Taiga, additional, Olin, Rebecca, additional, Olsson, Richard F., additional, Pawarode, Attaphol, additional, Peres, Edward, additional, Rezvani, Andrew R., additional, Rizzieri, David, additional, Savani, Bipin N., additional, Schouten, Harry C., additional, Sabloff, Mitchell, additional, Seftel, Matthew, additional, Seo, Sachiko, additional, Sorror, Mohamed L., additional, Szer, Jeff, additional, Wirk, Baldeep M., additional, Wood, William A., additional, and Artz, Andrew, additional

Published
2017
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143. Allogeneic Hematopoietic Cell Transplantation for Adult Chronic Myelomonocytic Leukemia

Author

Liu, Hien Duong, primary, Ahn, Kwang Woo, additional, Hu, Zhen-Huan, additional, Hamadani, Mehdi, additional, Nishihori, Taiga, additional, Wirk, Baldeep, additional, Beitinjaneh, Amer, additional, Rizzieri, David, additional, Grunwald, Michael R., additional, Sabloff, Mitchell, additional, Olsson, Richard F., additional, Bajel, Ashish, additional, Bredeson, Christopher, additional, Daly, Andrew, additional, Inamoto, Yoshihiro, additional, Majhail, Navneet, additional, Saad, Ayman, additional, Gupta, Vikas, additional, Gerds, Aaron, additional, Malone, Adriana, additional, Tallman, Martin, additional, Reshef, Ran, additional, Marks, David I., additional, Copelan, Edward, additional, Gergis, Usama, additional, Savoie, Mary Lynn, additional, Ustun, Celalettin, additional, Litzow, Mark R., additional, Cahn, Jean-Yves, additional, Kindwall-Keller, Tamila, additional, Akpek, Gorgun, additional, Savani, Bipin N., additional, Aljurf, Mahmoud, additional, Rowe, Jacob M., additional, Wiernik, Peter H., additional, Hsu, Jack W., additional, Cortes, Jorge, additional, Kalaycio, Matt, additional, Maziarz, Richard, additional, Sobecks, Ronald, additional, Popat, Uday, additional, Alyea, Edwin, additional, and Saber, Wael, additional

Published
2017
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145. Variable CD34+recovery of cryopreserved allogeneic HPC products: transplant implications during the COVID-19 pandemic

Author

Purtill, Duncan, Antonenas, Vicki, Chiappini, Paul, Tong, Daochen, O'Flaherty, Elizabeth, Bajel, Ashish, Kabani, Karieshma, Larsen, Stephen, Tan, Suikeat, Hutchins, Cheryl, Curtis, David J., Kennedy, Glen A., Watson, Anne-Marie, Bai, LiJun, Greenwood, Matthew, Gottlieb, David J., and Hamad, Nada

Abstract

Donor registries and transplantation societies recommend cryopreservation of unrelated donor hemopoietic progenitor cell (HPC) products before the recipient commences conditioning therapy to mitigate the donor and travel risks associated with the COVID-19 pandemic. However, little is known regarding the postthaw quality of such allogeneic products or the effect of precryopreservation storage and processing on these characteristics. We investigated the postthaw CD34+cell recovery and viability of 305 allogeneic HPC products cryopreserved at 9 laboratories across Australia. Median postthaw CD34+cell recovery was 76% and ranged from 6% to 122%. Longer transit time before cryopreservation, white cell count (WCC) during storage, and complex product manipulation before cryopreservation were independently associated with inferior postthaw CD34+cell recovery. Longer precryopreservation transit time and WCC were also associated with inferior postthaw CD34+cell viability. We conclude that although postthaw CD34+cell recovery and viability of cryopreserved allogeneic HPC is generally acceptable, there is a significant risk of poor postthaw product quality, associated with prolonged storage time, higher WCC, and complex product manipulation precryopreservation. Awareness of expected postthaw recovery and practices that influence it will assist collection, processing, and transplant centers in optimizing outcomes for transplant recipients.

Published
2020
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146. Variable CD34+ recovery of cryopreserved allogeneic HPC products: transplant implications during the COVID-19 pandemic

Author

Purtill, Duncan, Antonenas, Vicki, Chiappini, Paul, Tong, Daochen, O’Flaherty, Elizabeth, Bajel, Ashish, Kabani, Karieshma, Larsen, Stephen, Tan, Suikeat, Hutchins, Cheryl, Curtis, David J., Kennedy, Glen A., Watson, Anne-Marie, Bai, LiJun, Greenwood, Matthew, Gottlieb, David J., and Hamad, Nada

Abstract

Donor registries and transplantation societies recommend cryopreservation of unrelated donor hemopoietic progenitor cell (HPC) products before the recipient commences conditioning therapy to mitigate the donor and travel risks associated with the COVID-19 pandemic. However, little is known regarding the postthaw quality of such allogeneic products or the effect of precryopreservation storage and processing on these characteristics. We investigated the postthaw CD34+ cell recovery and viability of 305 allogeneic HPC products cryopreserved at 9 laboratories across Australia. Median postthaw CD34+ cell recovery was 76% and ranged from 6% to 122%. Longer transit time before cryopreservation, white cell count (WCC) during storage, and complex product manipulation before cryopreservation were independently associated with inferior postthaw CD34+ cell recovery. Longer precryopreservation transit time and WCC were also associated with inferior postthaw CD34+ cell viability. We conclude that although postthaw CD34+ cell recovery and viability of cryopreserved allogeneic HPC is generally acceptable, there is a significant risk of poor postthaw product quality, associated with prolonged storage time, higher WCC, and complex product manipulation precryopreservation. Awareness of expected postthaw recovery and practices that influence it will assist collection, processing, and transplant centers in optimizing outcomes for transplant recipients.

Published
2020
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147. Reduced intensity conditioning for acute myeloid leukemia using melphalan- vs busulfan-based regimens: a CIBMTR report

Author

Zhou, Zheng, Nath, Rajneesh, Cerny, Jan, Wang, Hai-Lin, Zhang, Mei-Jie, Abdel-Azim, Hisham, Agrawal, Vaibhav, Ahmed, Gulrayz, Al-Homsi, A. Samer, Aljurf, Mahmoud, Alkhateeb, Hassan B., Assal, Amer, Bacher, Ulrike, Bajel, Ashish, Bashir, Qaiser, Battiwalla, Minocher, Bhatt, Vijaya Raj, Byrne, Michael, Cahn, Jean-Yves, Cairo, Mitchell, Choe, Hannah, Copelan, Edward, Cutler, Corey, Damlaj, Moussab B., DeFilipp, Zachariah, De Lima, Marcos, Diaz, Miguel Angel, Farhadfar, Nosha, Foran, James, Freytes, César O., Gerds, Aaron T., Gergis, Usama, Grunwald, Michael R., Gul, Zartash, Hamadani, Mehdi, Hashmi, Shahrukh, Hertzberg, Mark, Hildebrandt, Gerhard C., Hossain, Nasheed, Inamoto, Yoshihiro, Isola, Luis, Jain, Tania, Kamble, Rammurti T., Khan, Muhammad Waqas, Kharfan-Dabaja, Mohamed A., Kebriaei, Partow, Kekre, Natasha, Khera, Nandita, Lazarus, Hillard M., Liesveld, Jane L., Litzow, Mark, Liu, Hongtao, Marks, David I., Martino, Rodrigo, Mathews, Vikram, Mishra, Asmita, Murthy, Hemant S., Nagler, Arnon, Nakamura, Ryotaro, Nathan, Sunita, Nishihori, Taiga, Olin, Rebecca, Olsson, Richard F., Palmisiano, Neil, Patel, Sagar S., Patnaik, Mrinal M., Pawarode, Attaphol, Perales, Miguel-Angel, Politikos, Ioannis, Popat, Uday, Rizzieri, David, Sandmaier, Brenda M., Savani, Bipin N., Seo, Sachiko, Shah, Nirav N., Uy, Geoffrey L., Valcárcel, David, Verdonck, Leo F., Waller, Edmund K., Wang, Youjin, Weisdorf, Daniel, Wirk, Baldeep, Wong, Eric, Yared, Jean A., and Saber, Wael

Abstract

There is a lack of large comparative study on the outcomes of reduced intensity conditioning (RIC) in acute myeloid leukemia (AML) transplantation using fludarabine/busulfan (FB) and fludarabine/melphalan (FM) regimens. Adult AML patients from Center for International Blood and Marrow Transplant Research who received first RIC allo-transplant between 2001 and 2015 were studied. Patients were excluded if they received cord blood or identical twin transplant, total body irradiation in conditioning, or graft-versus-host disease (GVHD) prophylaxis with in vitro T-cell depletion. Primary outcome was overall survival (OS), secondary end points were leukemia-free survival (LFS), nonrelapse mortality (NRM), relapse, and GVHD. Multivariate survival model was used with adjustment for patient, leukemia, and transplant-related factors. A total of 622 patients received FM and 791 received FB RIC. Compared with FB, the FM group had fewer transplant in complete remission (CR), fewer matched sibling donors, and less usage of anti-thymocyte globulin or alemtuzumab. More patients in the FM group received marrow grafts and had transplantation before 2005. OS was significantly lower within the first 3 months posttransplant in the FM group (hazard ratio [HR] = 1.82, P < .001), but was marginally superior beyond 3 months (HR = 0.87, P = .05). LFS was better with FM compared with FB (HR = 0.89, P = .05). NRM was significantly increased in the FM group during the first 3 months of posttransplant (HR = 3.85, P < .001). Long-term relapse was lower with FM (HR = 0.65, P < .001). Analysis restricted to patients with CR showed comparable results. In conclusion, compared with FB, the FM RIC showed a marginally superior long-term OS and LFS and a lower relapse rate. A lower OS early posttransplant within 3 months was largely the result of a higher early NRM.

Published
2020
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148. Increasing use of allogeneic hematopoietic cell transplantation in patients aged 70 years and older in the United States

Author

Muffly, Lori, Pasquini, Marcelo C., Martens, Michael, Brazauskas, Ruta, Zhu, Xiaochun, Adekola, Kehinde, Aljurf, Mahmoud, Ballen, Karen K., Bajel, Ashish, Baron, Frederic, Battiwalla, Minoo, Beitinjaneh, Amer, Cahn, Jean-Yves, Carabasi, Mathew, Chen, Yi-Bin, Chhabra, Saurabh, Ciurea, Stefan, Copelan, Edward, D'Souza, Anita, Edwards, John, Foran, James, Freytes, Cesar O., Fung, Henry C., Gale, Robert Peter, Giralt, Sergio, Hashmi, Shahrukh K., Hildebrandt, Gerhard C., Ho, Vincent, Jakubowski, Ann, Lazarus, Hillard, Luskin, Marlise R., Martino, Rodrigo, Maziarz, Richard, McCarthy, Philip, Nishihori, Taiga, Olin, Rebecca, Olsson, Richard F., Pawarode, Attaphol, Peres, Edward, Rezvani, Andrew R., Rizzieri, David, Savani, Bipin N., Schouten, Harry C., Sabloff, Mitchell, Seftel, Matthew, Seo, Sachiko, Sorror, Mohamed L., Szer, Jeff, Wirk, Baldeep M., Wood, William A., Artz, Andrew, Muffly, Lori, Pasquini, Marcelo C., Martens, Michael, Brazauskas, Ruta, Zhu, Xiaochun, Adekola, Kehinde, Aljurf, Mahmoud, Ballen, Karen K., Bajel, Ashish, Baron, Frederic, Battiwalla, Minoo, Beitinjaneh, Amer, Cahn, Jean-Yves, Carabasi, Mathew, Chen, Yi-Bin, Chhabra, Saurabh, Ciurea, Stefan, Copelan, Edward, D'Souza, Anita, Edwards, John, Foran, James, Freytes, Cesar O., Fung, Henry C., Gale, Robert Peter, Giralt, Sergio, Hashmi, Shahrukh K., Hildebrandt, Gerhard C., Ho, Vincent, Jakubowski, Ann, Lazarus, Hillard, Luskin, Marlise R., Martino, Rodrigo, Maziarz, Richard, McCarthy, Philip, Nishihori, Taiga, Olin, Rebecca, Olsson, Richard F., Pawarode, Attaphol, Peres, Edward, Rezvani, Andrew R., Rizzieri, David, Savani, Bipin N., Schouten, Harry C., Sabloff, Mitchell, Seftel, Matthew, Seo, Sachiko, Sorror, Mohamed L., Szer, Jeff, Wirk, Baldeep M., Wood, William A., and Artz, Andrew

Abstract

In this study, we evaluated trends and outcomes of allogeneic hematopoietic cell transplantation (HCT) in adults >= 70 years with hematologic malignancies across the United States. Adults >= 70 years with a hematologic malignancy undergoing first allogeneic HCT in the United States between 2000 and 2013 and reported to the Center for International Blood and Marrow Transplant Research were eligible. Transplant utilization and transplant outcomes, including overall survival (OS), progression-free survival (PFS), and transplant-related mortality (TRM) were studied. One thousand one hundred and six patients >= 70 years underwent HCT across 103 transplant centers. The number and proportion of allografts performed in this population rose markedly over the past decade, accounting for 0.1% of transplants in 2000 to 3.85% (N = 298) in 2013. Acute myeloid leukemia and myelodysplastic syndromes represented the most common disease indications. Two-year OS and PFS significantly improved over time (OS: 26% [95% confidence interval (CI), 21% to 33%] in 2000-2007 to 39% [95% CI, 35% to 42%] in 2008-2013, P < .001; PFS: 22% [16% to 28%] in 2000-2007 to 32% [95% CI, 29% to 36%] in 2008-2013, P = .003). Two-year TRM ranged from 33% to 35% and was unchanged over time (P = .54). Multivariable analysis of OS in the modern era of 2008-2013 revealed higher comorbidity by HCT comorbidity index >= 3 (hazard ratio [HR], 1.27; P = .006), umbilical cord blood graft (HR, 1.97; P = .0002), and myeloablative conditioning (HR, 1.61; P = .0002) as adverse factors. Over the past decade, utilization and survival after allogeneic transplant have increased in patients >= 70 years. Select adults >= 70 years with hematologic malignancies should be considered for transplant.

Published
2017
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149. Allogeneic Hematopoietic Cell Transplantation for Adult Chronic Myelomonocytic Leukemia

Author

Liu, Hien Duong, Ahn, Kwang Woo, Hu, Zhen-Huan, Hamadani, Mehdi, Nishihori, Taiga, Wirk, Baldeep, Beitinjaneh, Amer, Rizzieri, David, Grunwald, Michael R., Sabloff, Mitchell, Olsson, Richard F., Bajel, Ashish, Bredeson, Christopher, Daly, Andrew, Inamoto, Yoshihiro, Majhail, Navneet, Saad, Ayman, Gupta, Vikas, Gerds, Aaron, Malone, Adriana, Tallman, Martin, Reshef, Ran, Marks, David I., Copelan, Edward, Gergis, Usama, Savoie, Mary Lynn, Ustun, Celalettin, Litzow, Mark R., Cahn, Jean-Yves, Kindwall-Keller, Tamila, Akpek, Gorgun, Savani, Bipin N., Aljurf, Mahmoud, Rowe, Jacob M., Wiernik, Peter H., Hsu, Jack W., Cortes, Jorge, Kalaycio, Matt, Maziarz, Richard, Sobecks, Ronald, Popat, Uday, Alyea, Edwin, Saber, Wael, Liu, Hien Duong, Ahn, Kwang Woo, Hu, Zhen-Huan, Hamadani, Mehdi, Nishihori, Taiga, Wirk, Baldeep, Beitinjaneh, Amer, Rizzieri, David, Grunwald, Michael R., Sabloff, Mitchell, Olsson, Richard F., Bajel, Ashish, Bredeson, Christopher, Daly, Andrew, Inamoto, Yoshihiro, Majhail, Navneet, Saad, Ayman, Gupta, Vikas, Gerds, Aaron, Malone, Adriana, Tallman, Martin, Reshef, Ran, Marks, David I., Copelan, Edward, Gergis, Usama, Savoie, Mary Lynn, Ustun, Celalettin, Litzow, Mark R., Cahn, Jean-Yves, Kindwall-Keller, Tamila, Akpek, Gorgun, Savani, Bipin N., Aljurf, Mahmoud, Rowe, Jacob M., Wiernik, Peter H., Hsu, Jack W., Cortes, Jorge, Kalaycio, Matt, Maziarz, Richard, Sobecks, Ronald, Popat, Uday, Alyea, Edwin, and Saber, Wael

Abstract

Allogeneic hematopoietic cell transplantation (HCT) is potentially curative for patients with chronic myelomonocytic leukemia (CMML); however, few data exist regarding prognostic factors and transplantation outcomes. We performed this retrospective study to identify prognostic factors for post-transplantation outcomes. The CMML-specific prognostic scoring system (CPSS) has been validated in subjects receiving nontransplantation therapy and was included in our study. From 2001 to 2012, 209 adult subjects who received HCT for CMML were reported to the Center for International Blood and Marrow Transplant Research. The median age at transplantation was 57 years (range, 23 to 74). Median follow-up was 51 months (range, 3 to 122). On multivariate analyses, CPSS scores, Karnofsky performance status (KPS), and graft source were significant predictors of survival (P = .004, P = .01, P = .01, respectively). Higher CPSS scores were not associated with disease-free survival, relapse, or transplantation-related mortality. In a restricted analysis of subjects with relapse after HCT, those with intermediate-2/high risk had a nearly 2-fold increased risk of death after relapse compared to those with low/intermediate-1 CPSS scores. Respective 1-year, 3-year, and 5-year survival rates for low/intermediate-1 risk subjects were 61% (95% confidence interval [CI], 52% to 72%), 48% (95% CI, 37% to 59%), and 44% (95% CI, 33% to 55%), and for intermediate-2/high risk subjects were 38% (95% CI, 28% to 49%), 32% (95% CI, 21% to 42%), and 19% (95% CI, 8% to 29%). We conclude that higher CPSS score at time of transplantation, lower KPS, and a bone marrow graft are associated with inferior survival after HCT. Further investigation of CMML disease-related biology may provide insights into other risk factors predictive of post-transplantation outcomes. (C) 2017 American Society for Blood and Marrow Transplantation.

Published
2017
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150. The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry.

Author

Fox, Lucy C., McQuilten, Zoe K., Firkin, Frank, Fox, Vanessa, Badoux, Xavier, Bajel, Ashish, Barbaro, Pasquale, Cole-Sinclair, Merrole F., Forsyth, Cecily, Gibson, John, Hiwase, Devendra K., Johnston, Anna, Mills, Anthony, Roncolato, Fernando, Sutherland, Robyn, Szer, Jeff, Ting, Stephen B., Vilcassim, Shahla, Young, Lauren, and Waters, Neil A.

Abstract

The bone marrow failure syndromes (BMFS) are a diverse group of acquired and inherited diseases which may manifest in cytopenias, haematological malignancy and/or syndromic multisystem disease. Patients with BMFS frequently experience poor outcomes, and improved treatment strategies are needed. Collation of clinical characteristics and patient outcomes in a national disease-specific registry represents a powerful tool to identify areas of need and support clinical and research collaboration. Novel treatment strategies such as gene therapy, particularly in rare diseases, will depend on the ability to identify eligible patients alongside the molecular genetic features of their disease that may be amenable to novel therapy. The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry (AAR) aims to improve outcomes for all paediatric and adult patients with BMFS in Australia by describing the demographics, treatments (including supportive care) and outcomes, and serving as a resource for research and practice improvement. [ABSTRACT FROM AUTHOR]

Published
2023
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