What Does the Economic Burden of Acute Myeloid Leukemia Treatment Look Like for the Next Decade? An Analysis of Key Findings, Challenges and Recommendations

Figures & data

Table 1 Ongoing Trials of CAR-T Therapies in AML

Interventions	Study Phase	Estimated N	NCT Number
CD19 CAR-T	2/3	10	NCT04257175
CD123/CLL1 CAR- T	2/3	20	NCT03631576
CD123 CAR-T	1/2	45	NCT04265963
CD123 CAR-T	1/2	40	NCT04272125
CD123 CAR-T	1	12	NCT04678336
CD123 CAR-T	1	12	NCT03766126
CD123 UCAR-T	1	59	NCT03190278
CD38 CAR-T	1/2	20	NCT04351022
CD33 CAR-T	1/2	34	NCT03971799
CLL-1 CAR T	1	18	NCT04219163

Notes: Source: ClinicalTrials.gov, accessed 31 January 2021.

Abbreviations: AML, acute myeloid leukemia; CAR-T, chimeric antigen receptor T-cell; NCT, National Clinical Trial; UCAR-T, universal chimeric antigen receptor T-cell.

Table 2 Ongoing Phase 2 and Phase 3 Trials of Therapies Targeting Biologically or Genetically Defined Patient Subgroups or Molecular Pathways in AML

Target Sub-Population	Treatment	AML Treatment Phase	NCT Number
FLT-3	Crenolanib	Maintenance	NCT02400255; NCT03258931
		RR AML	NCT03250338
	Crenolanib, Midostaurin	ND AML	NCT03258931
	Gilteritinib, Midostaurin	ND AML	NCT03836209; NCT03836209
	Gilteritinib	ND AML	NCT02752035; NCT04293562; NCT02997202
		Maintenance	NCT02927262
	Midostaurin	ND AML	NCT03379727; NCT03280030
	Quizartinib	ND and maintenance	NCT02668653
		ND AML	NCT04676243; NCT04209725
	Ibrutinib	RR AML	NCT03642236
	ASP2215	RR AML	NCT03182244; NCT02421939
	SKLB1028	RR AML	NCT04716114; NCT04015024
IDH-1/IDH-2	Ivosidenib/enasidenib	ND IC AML	NCT03839771; NCT04493164
	Enasidenib	RR AML	NCT04203316
	Ivosidenib	ND NIC AML	NCT03173248
	AG-221 (CC-90007)	RR AML	NCT02577406
	Olaparib	RR AML	NCT03953898
NPM1	Gemtuzumab ozogamicin	ND IC AML	NCT00893399
	Pembrolizumab	RR AML	NCT03769532
	Oral arsenic trioxide	AML	NCT04689815
	SNDX-5613	RR AML	NCT04065399
CEBPA	HAD	ND IC AML	NCT04415008
HOX overexpression	Nintedanib	ND IC AML	NCT02665143
TP53	Decitabine	RR AML	NCT03063203
	Magrolimab	ND AML	NCT04778397
	APR-246 (eprenetapopt)	Maintenance	NCT03931291
CD33	Gemtuzumab ozogamicin	RR AML	NCT03839446
CD123+ or BPDCN-IPh-like+	Tagraxofusp	RR AML	NCT04342962
PD-1	Nivolumab	ND and RR AML	NCT02397720
		AML in remission	NCT02275533
		Maintenance	NCT02532231
		NIC RR AML	NCT03825367
	Visilizumab, azacitidine	RR AML	NCT04722952
	Tislelizumab + HMA	ND NIC AML	NCT04541277
	Pembrolizumab + decitabine	ND NIC and RR	NCT03969446
	Pembrolizumab + azacitidine	RR AML	NCT02845297
	Pembrolizumab + chemo	ND AML	NCT04214249
	Pembrolizumab + azacitidine +venetoclax	ND NIC AML	NCT04284787
	Pembrolizumab	AML in remission	NCT02771197
	Camrelizumab (SHR-1210)	RR AML	NCT04353479
	Visilizumab	RR AML	NCT04722952
RARA pathway associated biomarker	SY-1425	AML	NCT02807558
TET2 mutations	Azacitidine	AML	NCT03397173

Note: Source: ClinicalTrials.gov, accessed 31 January 2021.

Abbreviations: AML, acute myeloid leukemia; BPDCN-IPh, blastic plasmacytoid dendritic cell neoplasm-like phenotype; CEBPA, CCAAT enhancer-binding protein alpha; FLT-3, fms-like tyrosine kinase 3; HAD, homoharringtonine + cytarabine + daunorubicin; HMA, hypomethylating agent; HOX, homeobox; IC, intensive chemotherapy; IDH, isocitrate dehydrogenase; NCT, National Clinical Trial; ND, newly diagnosed; NIC, non-intensive chemotherapy; NPM1, nucleophosmin-1; PD-1, programmed cell death-1; RARA, retinoic acid receptor alpha; RR, relapsed refractory; TET2, Tet methylcytosine dioxygenase 2; TP53, tumor protein 53.

Table 3 Notable Ongoing Phase 2 and Phase 3 Studies in RR AML with Primary Results Expected by 2023

Intervention	Study Phase	Estimated N	Primary Completion Date	NCT Number
Uproleselan (GMI-1271) + IC	3	380	2022	NCT03616470
Crenolanib + IC	3	322	2021	NCT03250338
^131Iapamistamab (Iomab-B)	3	150	2021	NCT02665065
Gilteritinib	3	318	2021	NCT03182244
Ibrutinib + IC ± sorafenib	2/3	122	2022	NCT03642236
Alvocidib	2	134	2022	NCT03969420
Cladribine + idarubicin + cytarabine + quizartinib	2	86	2022	NCT04047641
Isatuximab + IC	2	96	2021	NCT03860844
Liposomal daunorubicin-cytarabine + venetoclax	2	52	2021	NCT03629171
Liposomal daunorubicin-cytarabine + gemtuzumab ozogamicin	2	50	2022	NCT03672539
Nivolumab + azacitidine ± ipilimumab	2	182	2022	NCT02397720
Olaparib	2	94	2022	NCT03953898
Pembrolizumab + azacitidine	2	67	2021	NCT02845297
Venetoclax + decitabine	2	400	2023	NCT03404193

Note: Source: ClinicalTrials.gov, accessed 31 January 2021.

Abbreviations: AML, acute myeloid leukemia; IC, intensive chemotherapy; NCT, National Clinical Trial.