Clinicians’ Perspectives on Cure in Adult Patients with Acute Lymphoblastic Leukemia with Minimal Residual Disease: A Delphi Study

Gidman, W; Shah, S; Zhang, L; McKendrick, J; Cong, Z; Cohan, D; Ottmann, O

Clinicians’ Perspectives on Cure in Adult Patients with Acute Lymphoblastic Leukemia with Minimal Residual Disease: A Delphi Study

Gidman, W Shah, S Zhang, L McKendrick, J

Cong, Z Cohan, D Ottmann, O

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Publication Type:: Journal Article
Citation:: Advances in Therapy, 2019, 36 (11), pp. 3017 - 3029
Issue Date:: 2019-11-01

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Field	Value	Language
dc.contributor.author	Gidman, W	en_US
dc.contributor.author	Shah, S	en_US
dc.contributor.author	Zhang, L	en_US
dc.contributor.author	McKendrick, J https://orcid.org/0000-0002-3527-834X	en_US
dc.contributor.author	Cong, Z	en_US
dc.contributor.author	Cohan, D	en_US
dc.contributor.author	Ottmann, O	en_US
dc.date.available	2020-11-30T18:12:36Z
dc.date.issued	2019-11-01	en_US
dc.identifier.citation	Advances in Therapy, 2019, 36 (11), pp. 3017 - 3029	en_US
dc.identifier.issn	0741-238X	en_US
dc.identifier.uri	http://hdl.handle.net/10453/136830
dc.description.abstract	© 2019, The Author(s). Hematologic complete remission (CR) is achievable for most adults with B cell precursor acute lymphoblastic leukemia (BCP-ALL). However, minimal residual disease (MRD) in patients with hematologic CR is associated with increased risk of relapse, shorter survival, and poorer transplantation outcomes. This study explored the concept of cure in adults with Philadelphia chromosome-negative (Ph−) BCP-ALL by MRD status at first hematologic CR (CR1) to inform evaluation of the clinical and economic benefits of new agents, where the concept of cure is important but long-term data are not available. The study used modified Delphi methodology involving clinicians experienced in the treatment of adult ALL. Participants completed a questionnaire, which was followed by country-specific panel discussions to discuss results and identify consensus on concepts and definitions. Clinicians from France (n = 4), Germany (n = 4), and the UK (n = 5) took part. Participants described cure in terms of the probability of future relapse. Relapse-free survival (RFS) was the preferred outcome measure to describe cure for the three patient groups considered (patients with MRD at CR1; patients who become negative for MRD after further treatment; patients who continue to have MRD). Consensus was reached on definitions of cure: that cure would begin to be considered at 3 years’ RFS and/or would be highly likely at 5 years’ RFS. Participants agreed that patients with MRD should usually undergo hematopoietic stem cell transplantation to have the best chance of survival; consensus was reached that alternatives are required when transplantation is not an option. Panels agreed that patients who achieve cure have a higher mortality rate and lower health-related quality of life than the general population. This study provides quantitative and qualitative information on the concept of cure in Ph− BCP-ALL in CR by MRD status applicable to interpreting the value of new therapies. Funding: Amgen. Plain Language Summary: Plain language summary available for this article.	en_US
dc.relation.ispartof	Advances in Therapy	en_US
dc.relation.isbasedon	10.1007/s12325-019-01099-x	en_US
dc.subject.classification	General Clinical Medicine	en_US
dc.title	Clinicians’ Perspectives on Cure in Adult Patients with Acute Lymphoblastic Leukemia with Minimal Residual Disease: A Delphi Study	en_US
dc.type	Journal Article
utslib.citation.volume	11	en_US
utslib.citation.volume	36	en_US
utslib.for	1112 Oncology and Carcinogenesis	en_US
utslib.for	1115 Pharmacology and Pharmaceutical Sciences	en_US
pubs.embargo.period	Not known	en_US
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Health
utslib.copyright.status	open_access	*
pubs.issue	11	en_US
pubs.publication-status	Published	en_US
pubs.volume	36	en_US

Abstract:

© 2019, The Author(s). Hematologic complete remission (CR) is achievable for most adults with B cell precursor acute lymphoblastic leukemia (BCP-ALL). However, minimal residual disease (MRD) in patients with hematologic CR is associated with increased risk of relapse, shorter survival, and poorer transplantation outcomes. This study explored the concept of cure in adults with Philadelphia chromosome-negative (Ph−) BCP-ALL by MRD status at first hematologic CR (CR1) to inform evaluation of the clinical and economic benefits of new agents, where the concept of cure is important but long-term data are not available. The study used modified Delphi methodology involving clinicians experienced in the treatment of adult ALL. Participants completed a questionnaire, which was followed by country-specific panel discussions to discuss results and identify consensus on concepts and definitions. Clinicians from France (n = 4), Germany (n = 4), and the UK (n = 5) took part. Participants described cure in terms of the probability of future relapse. Relapse-free survival (RFS) was the preferred outcome measure to describe cure for the three patient groups considered (patients with MRD at CR1; patients who become negative for MRD after further treatment; patients who continue to have MRD). Consensus was reached on definitions of cure: that cure would begin to be considered at 3 years’ RFS and/or would be highly likely at 5 years’ RFS. Participants agreed that patients with MRD should usually undergo hematopoietic stem cell transplantation to have the best chance of survival; consensus was reached that alternatives are required when transplantation is not an option. Panels agreed that patients who achieve cure have a higher mortality rate and lower health-related quality of life than the general population. This study provides quantitative and qualitative information on the concept of cure in Ph− BCP-ALL in CR by MRD status applicable to interpreting the value of new therapies. Funding: Amgen. Plain Language Summary: Plain language summary available for this article.

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