Are there any studies regarding prophylactic tocilizumab for the prevention of CRS with BiTE therapy. Specifically with the following medications: tarlatumab, talquetamab, or teclistamab?

Comment by InpharmD Researcher

The quality and availability of data regarding the efficacy and safety of tocilizumab for preventing cytokine release syndrome (CRS) in patients receiving bispecific T-cell engager (BiTE) antibodies are limited, and guidance on tocilizumab use for BiTE immune-related adverse events are primarily derived from CAR-T therapy data. Although data specific to tocilizumab prophylaxis for patients receiving tarlatamab and talquetamab are lacking, studies assessing its use in teclistamab therapy suggest that administering tocilizumab before teclistamab reduces the incidence of CRS without additional safety concerns or impact on treatment response (see Tables 1-2).

Background

The 2020 Society for Immunotherapy of Cancer (SITC) clinical practice guidelines for the management of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) recommend consideration of tocilizumab early in the course of CRS for older adult patients or patients with extensive comorbidities, as well as adult patients who develop American Society for Transplantation and Cellular Therapy (ASTCT) grade 2 CRS. Tocilizumab may be administered in pediatric patients who develop prolonged ASTCT grade 2 CRS or an intolerance to fever. Notably, panel recommendations are not specific to patients with CRS on bispecific T cell engager (BiTE) therapy; patients who have previously undergone BiTE therapy may be treated with chimeric antigen receptor (CAR) T-cells, depending on individual expression of target antigens. There is no discussion regarding the efficacy or safety of tocilizumab in this patient subgroup. [1]

The 2022 guidelines from the American Society of Clinical Oncology (ASCO) regarding the management of immune-related adverse events in CAR T-cell therapy, including CRS and ICANS, suggest the use of tocilizumab, with or without corticosteroids, for the treatment of severe or prolonged CRS associated with CAR T-cell therapy. Tocilizumab use for the prevention of CRS is not described. Additionally, the guidelines note there is limited experience regarding other therapies and specific guidance for BiTE therapy is not provided. [2]

According to a 2023 consensus report from the European Myeloma Network on the prevention and management of adverse events during treatment with bispecific antibodies and CAR T-cells in multiple myeloma, tocilizumab may be used to effectively manage CRS; incidence and severity of CRS related to BiTE administration may be diminished with tocilizumab premedication, stepwise dosing, dexamethasone premedication, or temporary drug discontinuation, although data were not provided to corroborate tocilizumab efficacy. If a fever develops within 72 hours and there is no improvement with symptomatic treatments, a 60-minute infusion of tocilizumab (8 mg/kg) is suggested, with corticosteroids administered to patients with persistent (>3 days) or high-grade symptoms and patients with symptoms refractory to tocilizumab. There is no discussion regarding the safety of tocilizumab in this patient subgroup. [3], [4]

Additionally, a 2020 review article discussed the use of tocilizumab in the management of CRS, with consideration towards its place in therapy in the management of CRS. Based on the results of a retrospective analysis of data from prospective clinical trials conducted in patients who developed CRS after treatment with tisagenlecleucel (CTL019) and axicabtagene ciloleucel (KTE-C19, axi-cel), tocilizumab is approved for the treatment of severe or life-threatening CAR T cell-induced CRS in adults and pediatrics (aged ≥ 2 years). To avoid altering the efficacy of CAR-T-cell therapy, the general consensus suggests administering tocilizumab at the time of moderate to severe signs of CRS. In the absence of clinical improvement, tocilizumab administration may be repeated every 8 hours to a maximum of four doses. Concomitant administration of steroids may be considered if no improvement is observed with the first dose of tocilizumab. While rare cases of CRS refractory to tocilizumab are reported, the mechanism of failure is unclear; however, it could be related to inadequate tocilizumab dosing to suppress IL-6 cytokines, alternative cytokines resulting in hypercytokinemia, or compensatory feedback in IL-6 signaling. The efficacy of tocilizumab is well-established ​​in the management of CAR-T-associated CRS without suppressing T-cell function and/or inducing T-cell apoptosis. Future studies are warranted to demonstrate an optimal timing of administration, especially in patient populations with a higher risk of developing CRS. Until the published date of review, no evidence demonstrated the benefits of using tocilizumab for the prevention or treatment of toxicity due to ICANS. Although the potential for BiTE therapy including CRS is described within the review, the use of prophylactic tocilizumab specifically for this purpose is not addressed. [5]

A 2023 prospective study evaluated the use of prophylactic tocilizumab to reduce CRS incidence in patients treated with teclistamab. In the MajesTEC-1 trial, CRS occurred in 72.1% of patients receiving the recommended phase 2 dose of teclistamab (1.5 mg/kg weekly), prompting this investigation. In this study, 14 patients received subcutaneous teclistamab following two step-up doses, with tocilizumab administered as a single IV dose (8 mg/kg) within 4 hours before the first teclistamab dose. CRS occurred in 4 patients (29%), with no events reaching grade 3 or higher; the median time to onset and duration were both 2 days. All cases were managed effectively with tocilizumab, allowing uninterrupted teclistamab treatment. Seven patients had grade 3 or 4 neutropenia, consistent with the overall MajesTEC-1 population, and 2 experienced grade 3 or 4 infections, with 2 reporting neurotoxic adverse events. Among response-evaluable patients, 4 of 7 responded to treatment. The authors concluded that a single dose of tocilizumab before teclistamab treatment reduced CRS incidence compared to the MajesTEC-1 population, with no new safety signals and no impact on treatment response. However, only the abstract was available for scrutiny, limiting a comprehensive assessment. [6], [7]

References:

[1] Maus MV, Alexander S, Bishop MR, et al. Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immune effector cell-related adverse events. J Immunother Cancer. 2020;8(2):e001511. doi:10.1136/jitc-2020-001511
[2] Santomasso BD, Nastoupil LJ, Adkins S, et al. Management of Immune-Related Adverse Events in Patients Treated With Chimeric Antigen Receptor T-Cell Therapy: ASCO Guideline [published correction appears in J Clin Oncol. 2022 Mar 10;40(8):919]. J Clin Oncol. 2021;39(35):3978-3992. doi:10.1200/JCO.21.01992
[3] Ludwig H, Terpos E, van de Donk N, et al. Prevention and management of adverse events during treatment with bispecific antibodies and CAR T cells in multiple myeloma: a consensus report of the European Myeloma Network. Lancet Oncol. 2023;24(6):e255-e269. doi:10.1016/S1470-2045(23)00159-6
[4] Clynes RA, Desjarlais JR. Redirected T Cell Cytotoxicity in Cancer Therapy. Annu Rev Med. 2019;70:437-450. doi:10.1146/annurev-med-062617-035821
[5] Si S, Teachey DT. Spotlight on Tocilizumab in the Treatment of CAR-T-Cell-Induced Cytokine Release Syndrome: Clinical Evidence to Date. Ther Clin Risk Manag. 2020;16:705-714. Published 2020 Aug 4. doi:10.2147/TCRM.S223468
[6] van de Donk NWCJ, Garfall A, Benboubker L, et al. P911: EVALUATION OF PROPHYLACTIC TOCILIZUMAB (TOCI) FOR THE REDUCTION OF CYTOKINE RELEASE SYNDROME (CRS) TO INFORM THE MANAGEMENT OF PATIENTS (PTS) TREATED WITH TECLISTAMAB IN MAJESTEC-1. Hemasphere. 2023;7(Suppl ):e43149cb. Published 2023 Aug 8. doi:10.1097/01.HS9.0000970548.43149.cb
[7] Usmani SZ, Garfall AL, van de Donk NWCJ, et al. Teclistamab, a B-cell maturation antigen × CD3 bispecific antibody, in patients with relapsed or refractory multiple myeloma (MajesTEC-1): a multicentre, open-label, single-arm, phase 1 study. Lancet. 2021;398(10301):665-674. doi:10.1016/S0140-6736(21)01338-6

Literature Review

A search of the published medical literature revealed 2 studies investigating the researchable question:

Are there any studies regarding prophylactic tocilizumab for the prevention of CRS with BiTE therapy. Specifically with the following medications: tarlatumab, talquetamab, or teclistamab?

Level of evidence

C - Multiple studies with limitations or conflicting results  Read more→



Please see Tables 1-2 for your response.


Prophylactic tocilizumab to prevent cytokine release syndrome (CRS) with teclistamab: A single-center experience
Design

Single-center observational study

N= 53 

Objective

To evaluate the efficacy of prophylactic tocilizumab in reducing the incidence and severity of cytokine release syndrome (CRS) in patients receiving teclistamab for relapsed/refractory multiple myeloma (RRMM)

Study Groups

No prophylactic tocilizumab (n= 15)

Prophylactic tocilizumab (n= 38)

Inclusion Criteria

Patients with relapsed/refractory multiple myeloma who are refractory to immunomodulatory drugs (IMID), proteasome inhibitors (PI), and anti-CD38 monoclonal antibodies

Exclusion Criteria

Not explicitly stated   

Methods

Teclistamab was administered in step-up doses as suggested by the package insert, with doses of 0.06 mg/kg, 0.3 mg/kg, and 1.5 mg/kg given at least 48 hours apart, followed by the first full dose. Premedications, including dexamethasone (16 mg), diphenhydramine (25-50 mg), and acetaminophen (650 mg), were given 30 minutes prior to each dose.

In evaluating the first 15 patients, the median time to CRS from the administration of the first priming dose was found to be 48 hours. To mitigate this, tocilizumab was administered prophylactically at a dose of 8 mg/kg intravenously (IV) over one hour (with a maximum dose of 800 mg) at 44 hours (4 hours prior to the second step-up dose level) for the next 38 patients.

Cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome (ICANS) were graded according to the American Society for Transplantation and Cellular Therapy (ASTCT) criteria and managed according to institutional guidelines.

Duration

December 2022 to August 2023

Follow-up: median of 113 days (range: 3-254)

Outcome Measures

Incidence and severity of CRS

Baseline Characteristics   No Prophylactic Tocilizumab (n = 15)

Prophylactic Tocilizumab (n= 38) 

Median Age, years (range) 58 (47–73) 69 (43–83) 

Male

13 (86.7%) 25 (65.8%) 

Race

White

Black/African American

Hispanic

Asian

Other 

 

7 (46.7%)

8 (53.3%)

-

-

-

 

18 (47.4%)

16 (42.1%)

3 (7.8%)

1 (2.6%)

3 (7.9%)

 

ISS Stage at Diagnosis

I

II

III

 

2 (22.2%)

2 (22.2%)

5 (55.6%)

 

14 (36.8%)

8 (21.1%)

15 (39.5%)

 
High-Risk Cytogenetics 4 (26.7%)

17 (44.7%)

Median Prior Lines of Therapy, (range) 6 (4–14)

5 (2–13) 

Abbreviations: ISS= international staging system

Results Endpoint

No Prophylactic Tocilizumab (n = 15)

Prophylactic Tocilizumab (n = 38) 
CRS incidence 

11 (73.3%) 

10 (26.3%)*

CRS Grade

Grade 1

Grade 2

Grade 3

 

10 (66.7%)

0

0

 

8 (21.1%)

1 (2.63%)

1 (2.63%)

Median number of tocilizumab doses (range)

1 (1-3)

Concurrent ICANS present  20% 5.3% 

*5/10 patients experienced CRS after step-up dose 1 and received tocilizumab treatment rather than prophylaxis.

One patient in the prophylactic group with grade 3 CRS received one dose of steroids for treatment, in addition to premedication steroids. No other patients treated with prophylactic tocilizumab required steroids. 

The use of prophylactic tocilizumab resulted in responses comparable to those seen in clinical trials, with a 70% overall response rate (in patients with assessable responses) and 15 of the 30 assessable patients (50%) achieving a very good partial response (VGPR) or better. 

Tocilizumab did not increase the incidence of grade 3 or 4 neutropenia compared to clinical trial data (42.1% vs. 64.2% in MasjesTEC-1 trial). 

Adverse Events

See results

Study Author Conclusions

Prophylactic tocilizumab effectively reduces the incidence and severity of CRS in RRMM patients receiving teclistamab, allowing for safe outpatient administration without increasing severe neutropenia or impacting treatment response.   

Critique

The study demonstrates several strengths, including an effective reduction in the incidence and severity of CRS, response rates comparable to those observed in clinical trials, and no increase in severe neutropenia. However, it has limitations, such as being a single-center study, having a small sample size, and a relatively short follow-up period. Moreover, the findings were presented in an editorial rather than a full study, which limits the depth of analysis and interpretation.

References:

Scott SA, Marin EM, Maples KT, et al. Prophylactic tocilizumab to prevent cytokine release syndrome (CRS) with teclistamab: A single-center experience. Blood Cancer J. 2023;13(1):191. Published 2023 Dec 20. doi:10.1038/s41408-023-00963-y

Prophylactic tocilizumab reduces the incidence of cytokine release syndrome in relapsed/refractory myeloma patients treated with teclistamab: Implications for outpatient step‐up dosing
Design

Single-center study

N= 29 

Objective To evaluate the efficacy of tocilizumab administered prior to the first step‐up dose to prevent the development of cytokine‐release syndrome (CRS) following the initiation of teclistamab therapy
Study Groups Prophylactic tocilizumab (N= 29)
Inclusion Criteria

Patients with relapsed/refractory multiple myeloma; received teclistamab monotherapy

Exclusion Criteria

Oligosecretory disease, severe cytopenias, renal impairment, and cardiac conditions

Methods

Teclistamab was administered with two step-up doses (0.06 and 0.3 mg/kg) followed by a full dose of 1.5 mg/kg every week (48 to 72 hr between step‐up doses and the first full dose). In patients undergoing hemodialysis, teclistamab was given directly after hemodialysis sessions. Prophylactic tocilizumab (8 mg/kg intravenously [IV]; maximum dose of 800 mg) was administered 1 hour prior to the first step-up dose. Patients also received 16 mg dexamethasone, 2 mg clemastine, and 1000 mg acetaminophen 1 hr prior to both step‐up doses and the first full dose.

Duration

Data collection: October 2022 to March 2024

Follow-up: median 8.7 months 

Outcome Measures

Incidence of CRS

Baseline Characteristics Characteristics Prophylactic tocilizumab (N= 29)
Age, years (IQR) 62 (57 to 69)
Female 13 (45%)
Presence of extramedullary plasmacytomas 8 (28%)

Cytogenetic risk profile, n (%)

High risk

High-risk (extended)

Standard risk

Standard risk (extended)

 

10 (34%)

22 (76%)

19 (66%)

7 (24%)

Laboratory values at baseline (IQR)

Absolute neutrophil count, ×109/L

Hemoglobin level, mmol/L

Platelet count, ×109/L

 eGFR, mL/min/1.73 m2

 

2.67 (1.75 to 3.08)

6.70 (6.10 to 7.50)

168 (107 to 209)

80 (66 to 90)

Prior lines of treatment (IQR)

Autologous SCT

Allogeneic SCT

4 (2 to 10)

24 (83%)

2 (6.9%)

Results

Endpoint

Prophylactic tocilizumab (N= 29)

CRS Rate

3 (10.3%)

Tocilizumab prophylaxis had no negative impact on the activity of teclistamab with 24 of the 29 patients (82.8%) achieving a partial response or better with ≥ very good partial response (VGPR) in 75.9%. Overall survival at 12 months was 72.2%.

CRS occurred in only three of the 29 patients: one patient experienced grade 1 CRS after step-up dose 1, another had grade 2 CRS after step-up dose 1, and a third patient experienced grade 1 CRS following step-up dose 2 and grade 2 CRS after the first full dose. Treatment included tocilizumab for all three patients and dexamethasone (10 mg IV) for the patients with recurrent CRS. Both patients with grade 2 CRS received IV fluids due to hypotension. The median CRS duration was one day (range, 1–3), and all CRS events resolved completely.

Adverse Events

Grade ≥3 thrombocytopenia: 13.8%, Grade ≥3 anemia: 6.9%, Grade ≥3 infections: 27.6%, No immune effector cell‐associated neurotoxicity syndrome (ICANS) observed.

Study Author Conclusions

Prophylactic tocilizumab administered prior to step‐up dose 1 markedly reduced the frequency of CRS without affecting efficacy. Although step‐up dosing is commonly administered in an inpatient setting, we also demonstrate that the low CRS rate with tocilizumab prophylaxis may improve the safety and feasibility of outpatient teclistamab step‐up dosing with a low risk of admission to the hospital for CRS treatment.

Critique The small sample size limits the ability to draw definitive conclusions about predictive factors for CRS. The study also lacks a control group for comparison, making it difficult to determine whether the observed outcomes are due to the intervention itself or other external factors.
References:

Korst CLBM, Groen K, Bosman PWC, et al. Prophylactic tocilizumab reduces the incidence of cytokine release syndrome in relapsed/refractory myeloma patients treated with teclistamab: Implications for outpatient step-up dosing. Hemasphere. 2024;8(7):e132. Published 2024 Jul 24. doi:10.1002/hem3.132