Real-World Outcome in the pre-CAR-T Era of Myeloma Patients Qualifying for CAR-T Cell Therapy.

Brechbühl, Simon; Bacher, Ulrike; Jeker, Barbara; Pabst, Thomas (2021). Real-World Outcome in the pre-CAR-T Era of Myeloma Patients Qualifying for CAR-T Cell Therapy. Mediterranean journal of hematology and infectious diseases, 13(1), e2021012. Università Cattolica del Sacro Cuore 10.4084/MJHID.2021.012

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Background

CAR-T cell therapy is likely to be introduced starting from 2021 in patients with relapsed/refractory myeloma (r/r MM) in Europe. In order to qualify for commercial CAR-T treatment, it is assumed that r/r MM patients will have to be exposed to at least three lines of previous treatments including lenalidomide, bortezomib and anti-CD38 treatment. However, the outcome of this particular subgroup of r/r MM patients is largely unknown whereas this knowledge is crucial to estimate the possible benefit of eventual CAR-T treatment.

Methods

In this non-interventional, retrospective single-center study, we analyzed all subsequent r/r MM patients treated between 01/2016 (when anti-CD38 treatment was commercially introduced in Switzerland) and 04/2020 at the University Hospital of Bern. Patients were eligible for the study if they had received at least three lines of treatment including one proteasome inhibitor (PI), one immunomodulatory drug (IMID) and one anti-CD38 antibody, and if they were in need of subsequent treatment and effectively received further lines of treatment.

Results

Among 56 patients fulfilling the criteria of at least three lines of treatment including PI, IMID and anti-CD38 treatment, only 34 (60%) effectively received subsequent further therapy. This suggests that 40% of r/r MM patients never receive additional treatment after at least three lines of treatment including PI, IMID and anti-CD38 treatment. For patients receiving further treatment, the median number of previous lines of treatment was 4.5 (range 2-12), including autologous stem cell transplantation in 31 (91%) patients. 13 (37%) patients were penta-refractory. The most frequently used treatment options were IMID/dexamethasone treatment in 11 (32%) patients, followed by PI/dexamethasone in 10 (29%) patients. 21 (62%) patients received two or more additional lines of therapy. The median PFS was 6.6 months (range 0-36.6 months), the median TTNT was 7.5 months (range 1.4-24.5 months) and the median OS was 13.5 months, (range 0.1-38 months) for the first subsequent treatment. The overall response rate (ORR) to the first subsequent treatment was 41%, with a median duration of the response of 5 months (range 1-37 months). 12% of the patients achieved VGPR or better, with a median duration of response of 8 months (range 3-37 months).

Conclusions

Myeloma patients refractory after at least three lines of anti-CD38/PI/IMID treatment have a poor prognosis with a PFS of 6.6 months and OS of 13.5 months. These data may serve as reference to compare the potential benefit of CAR-T treatment in this group of myeloma patients when available in the near future.

Item Type:

Journal Article (Original Article)

Division/Institute:

04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Haematology and Central Haematological Laboratory
04 Faculty of Medicine > Department of Haematology, Oncology, Infectious Diseases, Laboratory Medicine and Hospital Pharmacy (DOLS) > Clinic of Medical Oncology

UniBE Contributor:

Bacher, Vera Ulrike; Jeker, Barbara and Pabst, Thomas Niklaus

Subjects:

600 Technology > 610 Medicine & health

ISSN:

2035-3006

Publisher:

Università Cattolica del Sacro Cuore

Language:

English

Submitter:

Pierrette Durand Lüthi

Date Deposited:

15 Feb 2021 14:54

Last Modified:

21 Feb 2021 03:01

Publisher DOI:

10.4084/MJHID.2021.012

PubMed ID:

33489051

Uncontrolled Keywords:

Candidates for CAR-T cell therapy Myeloma Pre-study Real-world assessment Survival

BORIS DOI:

10.48350/151886

URI:

https://boris.unibe.ch/id/eprint/151886

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