What evidence is available regarding the efficacy of Rebyota (fecal microbiota, live-jslm) for management of Clostridioides difficile infections (CDI)?

Comment by InpharmD Researcher

While guidelines have yet to reflect the recent approval of Rebyota (fecal microbiota rectal suspension) and its place of therapy, phase 2b and 3 trials have demonstrated the safety and efficacy of this novel agent in reducing recurrent C. difficile infection following standard-of-care antibiotics with a sustained response. As the first-in-class therapy and placebo-controlled trial design, direct and indirect comparisons to alternative agents or fecal microbiota transplantation are lacking.

Background

Per the American College of Gastroenterology (ACG), fecal microbiota transplantation (FMT) may be considered part of the treatment plan for patients with severe and fulminant Clostridioides difficile infections (CDI) refractory to antibiotic therapy, particularly, when patients are deemed poor surgical candidates (strong recommendation, low quality of evidence). The organization also recommends use of FMT to prevent second or further recurrence of CDI (strong recommendation, moderate quality of evidence). ACG recommends FMT to be administered via colonoscopy; however, this guidance was developed before the approval of Rebyota. Similarly, Infectious Diseases Society of America (IDSA) and Society for Healthcare Epidemiology of America (SHEA) recommend FMT only for patients with multiple recurrences of CDI who have failed appropriate antibiotic treatments. They emphasize appropriate screening of donor and donor fecal specimens, considering safety reports where recipients have become ill or died. This guidance predates Rebyota’s approval. [1], [2]

A recent review published in the American Journal of Gastroenterology in 2023 acknowledges RBX2660, now Rebyota (fecal microbiota-live jslm), as the first live biotherapeutic product (LBP) that provides uniformly sourced specimens from comprehensively screened donors and ensures a predictable safety and efficacy profile. Phase 2 and 3 trials (see Tables 1 and 2) have demonstrated the efficacy of Rebyota in improving sustained treatment responsiveness and long-term recurrence in patients with CDI. Use of Rebyota has been reported to be well-tolerated, with most gastrointestinal adverse events being mild to moderate in nature. Of note, patients with inflammatory bowel disease and irritable bowel syndrome were generally excluded from clinical trials to minimize confounding sources of diarrhea. Given its recent approval and the need for guideline updates, the authors proposed a list of use criteria for Rebyota elaborating on FDA-approved indication based on expert opinions and postulated risk factors (see Table 3). Despite the inherent differences between FMT and Rebyota, clinicians may still use the guideline recommendations for FMT to guide indications. For patients with several risk factors for recurrence, earlier commencement of Rebyota prior to a second recurrence may be reasonable in those with several risk factors for recurrence. In such a patient population, the natural regrowth of microbiota may have already been impaired, suggesting benefit from LBP supplementation. In practice, patient selection and treatment initiation should be individualized. While robust data are lacking, repeated Rebyota may be administered to those with a recurrence within 8 weeks of initial treatment. Given the low quality of evidence associated with FMT as treatment in patients with severe or fulminant CDI who are refractory to antimicrobial therapy and poor candidates for surgical intervention, further studies are required to elucidate the role of Rebyota as a novel agent for this challenging population. [3]

As with any new treatment, the authors stress the need for understanding the logistics of its usage, including shipment, handling, and administration in the facility or office, as specified in the product label and state regulations. If allowed, larger volume providers with a dedicated session and room for rectal administration along with patient scheduling may facilitate the smooth incorporation of Rebyota into practice sites. Overall, Rebyota provides a promising option in the settings of recurrent CDI and likely improves access to needed therapies as FMT becomes scarce. [3]

A 2023 cost-effectiveness analysis evaluated the cost-effectiveness of RBL compared to SOC for preventing rCDI using a Markov model developed in Microsoft Excel® from the perspective of a US third-party healthcare payer. Adult patients with ≥1 recurrence after CDI, completed oral antibiotics, or had severe CDI resulting in hospitalization within a year were included, while those with known rCDI history, IBD, IBS, chronic diarrhea, and previous FMT were excluded. The intervention involved RBL rectal suspension versus SOC after antibiotic therapy. RBL showed cost-effectiveness with an incremental cost-effectiveness ratio (ICER) of $18,727 per quality-adjusted life year (QALY) gained, $20,186 per life year gained versus SOC, and $13,727 per QALY gained for first recurrence patients. Improved QALYs, reduced healthcare resource utilization, and savings in medical costs contributed to RBL's cost-effectiveness. One limitation is that the study’s efficacy data was based on trials with a 24-month follow-up, and assumptions were made for long-term response, meaning real-world data is still needed. Nevertheless, investigators concluded that RBL is cost-effective compared to SOC at preventing rCDI with an ICER of $18,727 per QALY gained among patients with one or more rCDI. [4]

References:

[1] Kelly CR, Fischer M, Allegretti JR, et al. ACG Clinical Guidelines: Prevention, Diagnosis, and Treatment of Clostridioides difficile Infections [published correction appears in Am J Gastroenterol. 2022 Feb 1;117(2):358]. Am J Gastroenterol. 2021;116(6):1124-1147. doi:10.14309/ajg.0000000000001278
[2] Johnson S, Lavergne V, Skinner AM, et al. Clinical Practice Guideline by the Infectious Diseases Society of America (IDSA) and Society for Healthcare Epidemiology of America (SHEA): 2021 Focused Update Guidelines on Management of Clostridioides difficile Infection in Adults. Clin Infect Dis. 2021;73(5):e1029-e1044. doi:10.1093/cid/ciab549
[3] Feuerstadt P, Allegretti JR, Khanna S. Practical Use of Rebyota for the Prevention of Recurrent Clostridioides difficile Infection [published online ahead of print, 2023 Jan 25]. Am J Gastroenterol. 2023;10.14309/ajg.0000000000002195. doi:10.14309/ajg.0000000000002195
[4] Lodise T, Guo A, Yang M, et al. Cost-Effectiveness Analysis of REBYOTA™ (Fecal Microbiota, Live-jslm [FMBL]) Versus Standard of Care for the Prevention of Recurrent Clostridioides difficile Infection in the USA. Adv Ther. 2023;40(6):2784-2800. doi:10.1007/s12325-023-02505-1

Literature Review

A search of the published medical literature revealed 5 studies investigating the researchable question:

What evidence is available regarding the efficacy of Rebyota (fecal microbiota-live jslm) for management of Clostridioides difficile infections (CDI)?

Level of evidence

B - One high-quality study or multiple studies with limitations  Read more→



Please see Tables 1-5 for your response.


 

Final Results from a Phase 2b Randomized, Placebo-Controlled Clinical Trial of RBX2660: A Microbiota-Based Drug for the Prevention of Recurrent Clostridioides difficile Infection

Design

Prospective, multicenter, randomized, double-blinded, placebo-controlled, three-arm phase 2b study 

N= 133

Objective

To report the final safety data of PUNCH CD2 clinical trial through 24 months of follow-up as well as final efficacy data, reflecting alignment of the pre-specified statistical analysis plan definitions with the data presented

Study Groups

Final intention-to-treat (ITT) population (N= 133)

Group A (2 × RBX2660; aka Rebyota™) (n= 45)

Group B (2 × placebo) (n= 44)

Group C (1 × RBX2660, 1 × placebo) (n= 44)

Inclusion Criteria

At least 18 years of age and had at least three episodes of Clostridioides difficile infection (CDI) and at least two rounds of standard antibiotic treatment or had at least two episodes of severe CDI resulting in hospitalization

Exclusion Criteria

Ongoing or anticipated antibiotic therapy for a condition other than CDI, known or suspected causes of diarrhea other than CDI, a compromised immune system, a history of inflammatory bowel disease, or pregnancy

Methods

Eligible patients were randomized (1:1:1) to receive two doses of Rebyota™; two doses of placebo; or one dose of Rebyota™ plus one dose of placebo, all administered 7 ± 2 days apart. Participants who recurred within 8 weeks were eligible to receive up to two open-label RBX2660 doses.

Duration

Follow-up: 24 months

Outcome Measures

Primary: treatment success at 8 weeks (prevention of recurrence, defined as absence of diarrhea and no re-treatment for CDI any time after the first dose until 8 weeks after the second dose of the study treatment)

Secondary: adverse events (AEs)

Baseline Characteristics

Based on previously published interim ITT

Group A (2 × RBX2660) (n= 41)

Group B (2 × placebo)
(n= 44)

Group C (1 × RBX2660, 1 × placebo)
(n= 42)

Median age, years (range)

66 (24-89) 62 (19-92)  63 (18-88)

Female

25 (61%) 30 (68%) 24 (57%)

White

39 (95%) 42 (96%) 40 (95%)

Antibiotic used at screening

Vancomycin

Fidaxomicin

Metronidazole

None

 

38 (93%)

1 (2%)

2 (5%)

0

 

40 (91%)

2 (5%)

2 (5%)

0

 

37 (88%)

2 (5%)

2 (5%)

1 (2%)

Median number of CDI episodes (range)

4 (3-11)

3 (2-11) 4 (2-14)

Median duration of CDI episode, days (range)

15 (1-74) 15 (1-98) 14 (1-71)

Results

Endpoint

Group A (2 × RBX2660)

Group B (2 × placebo)

Group C (1 × RBX2660, 1 × placebo)

Treatment success

Interim ITT (N= 127)

Final ITT (N= 133)

Final modified ITT (N= 121)

Interim per protocol (PP) (N= 94)

Final PP (N= 83)

 

61.0% (25/41) 

55.6% (25/45)

62.5% (25/40)

71.0% (22/31)

75.0% (21/28)

 

45.5% (20/44)

43.2% (19/44)

44.2% (19/43)

52.9% (18/34)

58.1% (18/31)

 

66.7% (28/42)*

56.8% (25/44)

65.8% (25/38)

79.3% (23/29)*

87.5% (21/24)*

Final safety results

Group A (2 × RBX2660) (n= 42)

Group B (2 × placebo) (n= 44) Group C (1 × RBX2660, 1 × placebo) (n= 42)

Any TEAE

TEAE related to study drugs

TEAE related to rectal administration

TEAE related to CDI

TEAE related to pre-existing condition

313/34 (81.0%) 

55/14 (33.3%)

40/10 (23.8%)

73/17 (40.5%)

159/27 (64.3%)

290/38 (86.4%)

33/15 (34.1%)

30/17 (38.6%)

59/16 (36.4%)

98/27 (61.4%)

238/33 (78.6%)

14/8 (19.0%)

14/7 (16.7%)

50/15 (35.7%)

105/21 (50.0%)

*p< 0.05 compared to Group B (2 x placebo)

Adverse Events

See Results

Study Author Conclusions

While the phase 2b PUNCH CD2 clinical trial did not meet its pre-defined primary endpoint of treatment success at 8 weeks after two doses of RBX2660 vs. two doses of placebo, clinically meaningful data were obtained to justify proceeding with the single dose regimen in the phase 3 clinical trial, PUNCH CD3, now complete. To date, the cumulative data for RBX2660 demonstrate consistent efficacy and safety outcomes for reduction of CDI recurrence in adults.

InpharmD Researcher Critique

The final analysis failed to demonstrate a statistically significant treatment response in two doses of Rebyota™ compared to two doses of placebo. What authors claimed as “clinically meaningful data” were obtained from the single dose Rebyota™ regimen in the modified ITT and PP populations.



References:

Dubberke ER, Orenstein R, Khanna S, Guthmueller B, Lee C. Final Results from a Phase 2b Randomized, Placebo-Controlled Clinical Trial of RBX2660: A Microbiota-Based Drug for the Prevention of Recurrent Clostridioides difficile Infection. Infect Dis Ther. 2023;12(2):703-709. doi:10.1007/s40121-022-00744-3

 

Efficacy and Safety of RBX2660 in PUNCH CD3, a Phase III, Randomized, Double-Blind, Placebo-Controlled Trial with a Bayesian Primary Analysis for the Prevention of Recurrent Clostridioides difficile Infection

Design

Randomized, double-blind, placebo-controlled, phase III trial (PUNCH CD3 trial)

N= 267

Objective

To compare the safety and efficacy of RBX2660 (aka Rebyota™) with placebo in reducing rates of recurrent Clostridioides difficile infection (rCDI)

Study Groups

Placebo (n= 87)

RBX2660 (n= 180)

Inclusion Criteria

Adults (aged ≥ 18 years) with documentation of rCDI (defined as one or more recurrences after a primary episode) and a positive stool assay for C. difficile who had completed one or more rounds of standard-of-care antibiotic therapy or had two or more episodes of severe CDI resulting in hospitalization within the past year

Exclusion Criteria

Known history of refractory CDI, inflammatory bowel disease, irritable bowel syndrome, chronic diarrhea, celiac disease, colostomy, active colitis, continued diarrhea despite antibiotic therapy, required antibiotic therapy for another condition, or had previous fecal microbiota transplantation (FMT)

Methods

Eligible patients were randomized (2:1) to receive either a subsequent blinded, single-dose enema of RBX2660 or placebo (normal saline) rectally by a qualified and trained healthcare professional after a full course of antibiotic treatment for rCDI, with a 24-72 h washout period. In the event of treatment failure within the first 8 weeks of blinded treatment, participants were offered the opportunity to receive an open-label treatment of RBX2660.

Duration

Between July 2017 and February 2020

Follow-up: 6 months

Outcome Measures

Primary: treatment success, defined as the absence of CDI diarrhea within 8 weeks of study treatment

Secondary: sustained clinical response, defined as treatment success of the presenting CDI recurrence and no new CDI episodes for greater than 8 weeks through 6 months after completing a study treatment; adverse events (AEs)

Baseline Characteristics

 

Placebo
(n= 87)

RBX2660
(n= 180)

     

Age, years

60.0 (26–86) 64.0 (19–93)      

Female

60 (69.0%) 123 (68.3%)      

White

78 (89.7%) 168 (93.3%)      

Ethnicity

Not Hispanic or Latino

 

80 (92.0%)

 

168 (93.3%)

     

Geography region

Northern USA

Eastern USA

Southern USA

Western USA

Outside of USA

 

14 (16.1%)

8 (9.2%)

28 (32.2%)

11 (12.6%)

26 (29.9%)

 

31 (17.2%)

24 (13.3%)

48 (26.7%)

26 (14.4%)

51 (28.3%)

     

Randomization strata

Vancomycin alone

Vancomycin combination therapya

Fidaxomicin

Otherb 

 

78 (89.7%)

2 (2.3%)

5 (5.7%)

2 (2.3%)

 

157 (87.2%)

5 (2.8%)

12 (6.7%)

6 (3.3%)

     

ATLAS score for qualifying CDI episode

3.0 (2–8) 3.0 (2–8)      

CDI episodes before blinded treatment

 ≤ 3

> 3

 

59 (67.8%)

28 (32.2%)

 

111 (61.7%)

69 (38.3%)

     

CDI confirmation testing methodc

PCR positive

EIA for toxin A/B positive

GDH positive

Other 

 

65 (74.7%)

16 (18.3%)

10 (11.5%)

6 (6.9%) 

 

130 (72.2%)

50 (27.8%)

36 (20.0%)

9 (5.0%)

     

EIA enzyme immunoassay, GDH glutamate dehydrogenase, PCR polymerase chain reaction

aCombination antibiotics included metronidazole and/or fidaxomicin

bOther included various other antibiotics alone or in combination

cParticipants may have had more than one testing assay to confirm most recent qualifying CDI episode

Results

Analysis population

Trial

Model estimated treatment success, %

Treatment effect (95% confidence interval [CI] Posterior probabilityb

Placebo

RBX2660

Planned primary endpoint analysisa

Intention-to-treat (ITT)

Modified ITT (mITT)

 

PUNCH CD2

PUNCH CD3

58.10% 70.40% 12.3 (1.4-23.3) 0.986354

Matched populationsc

mITT

 

PUNCH CD2

PUNCH CD3

57.5% 70.60% 13.1 (2.3–24.0) 0.99136
 

Through 6 months after blinded treatment

Through 6 months after open-label treatment    
Blinded placebo
(n= 87)

Blinded RBX2660
(n= 180)

Blinded placebo, open-label RBX2660 (n= 24) Blinded RBX2660, open-label RBX2660 (n= 41)   

All AEs

39 (44.8%) 100 (55.6%) 14 (58.3%) 24 (58.5%)  

AEs by maximum severity

Mild

Moderate

Severe

Potentially life-threatening

 

9 (10.3%)

25 (28.7%)

5 (5.7%)

0

 

42 (23.3%)

47 (26.1%)

10 (5.6%)

1 (0.6%)d

 

6 (25.0%)

6 (25.0%)

1 (4.2%)

1 (4.2%)

 

8 (19.5%)

10 (24.4%)

5 (12.2%)

1 (2.4%)

 
Discontinued due to AE 0 1 (0.6%)d 0 2 (4.9%)  
Serious AEs 2 (2.3%)  7 (3.9%)  1 (4.2%)  5 (12.2%)  
Deaths  1 (0.6%)d  1 (2.4%)  

aBayesian primary analysis integrated data from a previous phase IIb study (PUNCH CD2 trial) in which 42 adults received one dose of Rebyota™ and one dose of placebo, and 44 adults received two doses of placebo. 

bPosterior probability of superiority threshold is > 0.975.

cPUNCH CD3 analysis population definitions were applied to PUNCH CD2, and matched populations (e.g., PUNCH CD2 mITT and PUNCH CD3 mITT) were then used to generate model-estimated treatment success rates.

dSame participant represented in each category

More than 90% of the participants who achieved treatment success at 8 weeks had sustained responses through 6 months in both the RBX2660 (90.6%) and the placebo (92.1%) groups. 

Adverse Events

See Results

Study Author Conclusions

RBX2660 is a safe and effective treatment to reduce recurrent C. difficile infection following standard-of-care antibiotics with a sustained response through 6 months.

InpharmD Researcher Critique

The trial experienced substantial patient dropouts (n= 22 pre-trial and n= 33 during trial). Discontinuation was similar between groups which suggest poor approval of drug delivery method. Additionally, the placebo response was higher than expected. Authors speculate this may be due to false positives from PCR assay and therefore study may have patients without CDI.



References:

Khanna S, Assi M, Lee C, et al. Efficacy and Safety of RBX2660 in PUNCH CD3, a Phase III, Randomized, Double-Blind, Placebo-Controlled Trial with a Bayesian Primary Analysis for the Prevention of Recurrent Clostridioides difficile Infection [published correction appears in Drugs. 2022 Nov 7;:]. Drugs. 2022;82(15):1527-1538. doi:10.1007/s40265-022-01797-x

 

Effect of Fecal Microbiota, Live-Jslm (REBYOTA [RBL]) on Health-Related Quality of Life in Patients With Recurrent Clostridioides difficile Infection: Results From the PUNCH CD3 Clinical Trial

Design

Secondary analysis of a randomized, double-blind, placebo-controlled phase 3 study (PUNCH CD3)

N= 185

Objective

To analyze the Cdiff32 health-related quality of life (HRQL) data collected in the PUNCH CD3 trial and to compare the HRQL in adults with rCDI between patients randomized to fecal microbiota, live-jslm (Rebyota [RBL]) and those randomized to placebo through 8 weeks after dosing

Study Groups

Rebyota (n= 128)

Placebo (n= 57)

Inclusion Criteria

Adults (aged ≥ 18 years) with documentation of rCDI (defined as one or more recurrences after a primary episode) and a positive stool assay for C. difficile who had completed one or more rounds of standard-of-care antibiotic therapy or had two or more episodes of severe CDI resulting in hospitalization within the past year

Exclusion Criteria

Known history of refractory CDI, inflammatory bowel disease, irritable bowel syndrome, chronic diarrhea, celiac disease, colostomy, active colitis, continued diarrhea despite antibiotic therapy, required antibiotic therapy for another condition, or had previous fecal microbiota transplantation (FMT)

Methods

Eligible patients were randomized (2:1) to receive either a subsequent blinded, single-dose enema of RBX2660 or placebo (normal saline) rectally by a qualified and trained healthcare professional after a full course of antibiotic treatment for rCDI, with a 24-72 h washout period. In the event of treatment failure within the first 8 weeks of blinded treatment, participants were offered the opportunity to receive an open-label treatment of RBX2660.

For secondary analysis, the as-observed data of the modified intention-to-treat (mITT) population of PUNCH CD3 was analyzed. The mITT population included all patients who successfully received blinded treatment but excluded those who withdrew before treatment, for whom treatment was attempted but not completed, or who discontinued before evaluation of treatment failure or success at week 8 if the reason for dropout was unrelated to CDI.

HRQL was measured using the 32-item Cdiff32, a validated, disease-specific survey that quantifies changes in the HRQL using a total and 3 domain scores (physical, mental, and social), comprised of 32 self-administered items about the impact of CDI in physical, mental, and social domains with 5-point Likert scale responses. The total and domain scores of Cdiff32 ranged from 0 (worst score) to 100 (best score). The survey was administered at baseline and weeks 1, 4, and 8 during the double-blind period. 

Duration

Trial: July 2017 to February 2020

Intervention: 8 weeks

Follow-up: 6 months

Outcome Measures

Cdiff32 total and domain scores

Baseline Characteristics

 

Placebo (n= 57)

Rebyota (n= 128)

 

Age, years

< 65 years

57 ± 16.4

66.7%

60.8 ± 16.7

52.3%

 

Female

71.9% 68.0%  

White

87.7%  93.0%  

Disease characteristics

No. of CDI episodes before blinded treatment

Prior hospitalization due to CDI

 

3.0 ± 1.1

15.8%

 

3.2 ± 1.1

12.5%

 

Antibiotics used at baseline

Vancomycin

Fidaxomicin

Other

 

91.2%

7.0%

1.8%

 

88.3%

7.8%

3.9%

 

Proton pump inhibitor use

24.6%

18.0%

 

Results

Endpoint

Difference in Cdiff32 score, point estimate*

95% confidence interval

p-value

Cdiff32 domain

Total

Physical

Mental

Social

 

7.2

6.6

8.3

6.5 

 

1.2 to 13.2

0.8 to 12.3

1.4 to 15.3

-0.6 to 13.6

 

< 0.05

< 0.05

< 0.05

0.08 

Results presented above for multivariable-adjusted analysis at week 8 comparing RBL vs. placebo. At baseline, Cdiff32 scores were similar between groups, and total scores increased significantly from baseline to week 8 in both arms (all p< 0.001). With the exception of mental score (59.4 ± 27.3 placebo vs. 66.3 ± 21.8 Rebyota; p< 0.05) between group comparisons were not significant for any domain until adjusted multivariable analysis was done. 

*The point estimates represent the improvement in Cdiff32 total or domain score in patients given RBL versus placebo.

Adverse Events

N/A

Study Author Conclusions

In conclusion, in this secondary analysis of a randomized clinical trial (PUNCH CD3), treatment with Rebyota, compared with placebo, was associated with more sustained and profound improvements in overall HRQL—specifically, in physical and mental health status. These findings indicated that live biotherapeutic products such as RBL can positively affect patients’ HRQL, providing comprehensive benefits in the treatment of rCDI.

InpharmD Researcher Critique

This was a secondary analysis of a large, phase 3 trial (summarized in a separate table). A more limited sample size of patients was included in this subanalysis. 



References:

Garey KW, Dubberke ER, Guo A, et al. Effect of Fecal Microbiota, Live-Jslm (REBYOTA [RBL]) on Health-Related Quality of Life in Patients With Recurrent Clostridioides difficile Infection: Results From the PUNCH CD3 Clinical Trial. Open Forum Infect Dis. 2023;10(8):ofad383. Published 2023 Jul 20. doi:10.1093/ofid/ofad383

Indications for Rebyota (RBX2660)*

Second recurrence (third episode) following standard of care antimicrobial

First recurrence in patients that are at high risk for future recurrences

  • Age > 65
  • Chronic proton pump inhibitor usage
  • Immunocompromised (e.g., chronic kidney disease, diabetes mellitus, active chemotherapy)
  • Likely future concomitant antimicrobial usage
  • Lives in skilled nursing facility
  • Severe underlying illness
  • Spends significant amount of time as an inpatient at the hospital
  • Lives in skilled nursing facility
Recurs within 8 weeks of receiving an initial treatment 
FDA Indication: REBYOTA is indicated for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for recurrent CDI.
*The indications from this chart are based upon expert opinion and not RBX2660 data supporting these specific risk factors. 
References:

Adapted from:
Feuerstadt P, Allegretti JR, Khanna S. Practical Use of Rebyota for the Prevention of Recurrent Clostridioides difficile Infection [published online ahead of print, 2023 Jan 25]. Am J Gastroenterol. 2023;10.14309/ajg.0000000000002195. doi:10.14309/ajg.0000000000002195

 

Retrospective Analysis of the Safety and Efficacy of Fecal Microbiota, Live-jslm (REBYOTATM) Administered Under Enforcement Discretion to Patients With Clostridioides difficile Infection

Design

Retrospective, observational, multicenter analysis

N= 94

Objective

To evaluate the safety and tolerability of fecal microbiota, live-jslm (RBL; REBYOTA™) through 6 months after treatment in patients who received RBL under enforcement discretion (ED)

Study Groups

Full cohort (N= 94)

Inclusion Criteria

Age ≥ 18 years, received RBL under ED

Exclusion Criteria

No exclusion criteria 

Methods

Patient data were retrospectively collected via chart review. A single treatment course of RBL ranged from 1 to 2 doses, based on clinician's discretion (determined by treating physician before intervention). Patients who experienced recurrent Clostridioides difficile (rCDI) after the first course may have received a second course of RBL (maximum of 4 doses: 2 treatment courses of 2 doses each).

The full analysis set (FAS) included all patients who received RBL during study period. The primary safety set (PSS) was comprised of a subgroup of patients who were RBL treatment-naive and had comprehensive medical records for 6 months after treatment. 

Duration

Treated between November 1, 2015 to September 30, 2019

Outcome Measures

Primary: treatment-emergent adverse events (TEAEs)

Secondary: treatment success (prevention of rCDI within 8 weeks of treatment), sustained response for 6 months after treatment

Baseline Characteristics

 

FAS (N= 94)

 

    

Age, years

≥ 65 years

59.8

44.7%

Female

72.3% 

CDI confirmed via laboratory test

89.4%

Comorbidities

GI and nonspecific inflammation and dysfunctional

Immune-mediated/autoimmune disorders

Irritable bowel syndrome

Microscopic colitis

Crohn's disease

Ulcerative colitis

PSS (n= 64)

70.3%

65.6%

21.9%

10.9%

7.8%

6.3%

 

Results

Endpoint

PSS (n= 64)

 

Events

Number patients (%)

Any TEAE

Related to RBL

Related to procedure

Severe and life-threatening

Leading to death

Serious

Of special interest*

New chronic conditions or worsening of preexisting conditions

 

144 

32

4

10

1

11

58

33

 

40 (62.5)

11 (17.2)

3 (4.7) 

5 (7.8)

1 (1.6)

8 (12.5)

22 (34.4)

15 (23.4)

Treatment success

1 dose (n= 24)

2 doses (n= 40)

53 (82.8%) 

20 (83.3%)

33 (82.5%)

 

Sustained clinical response x 6 months

1 dose (n= 24)

2 doses (n= 40)

47 of 53 (88.7%)

90.0%

87.9%

*Most commonly worsened diarrhea and abdominal cramping or pain (14.1% of patients, each)

Most TEAEs (93%) were mild to moderate in severity

Adverse Events

Common Adverse Events: GI disorders (45.3%)

Serious Adverse Events: Serious adverse events occurred in 12.5% of patients. Severe and life-threatening AEs were reported in 7.8% of patients (ileus, organ failure, failure to thrive, and major depression); none were considered to be related to RBL or procedure.

Percentage that Discontinued due to Adverse Events: N/A

Study Author Conclusions

This retrospective analysis is representative of real-world diagnostics and a patient population typically encountered in everyday practice. The findings reinforce the potential efficacy and safety of RBL in real-world populations with common rCDI comorbidities representative of clinical practice. Future prospective studies with broad eligibility criteria across multiple centers may provide additional information on the efficacy and safety of RBL administration, as well as allow for comparative outcomes achieved with 1 and 2 doses, in the prevention of rCDI in real-world patient populations.

InpharmD Researcher Critique

This study is limited by its retrospective study design, but allowed for broad inclusion of all patients who received RBL under ED at the included clinical sites. A laboratory diagnosis of CDI was not a requirement for inclusion. 



References:

Feuerstadt P, Harvey A, Yoho DS, Garcia-Diaz JB, Knapple WL, Bancke L. Retrospective Analysis of the Safety and Efficacy of Fecal Microbiota, Live-jslm (REBYOTATM) Administered Under Enforcement Discretion to Patients With Clostridioides difficile Infection. Open Forum Infect Dis. 2023;10(5):ofad171. Published 2023 Mar 31. doi:10.1093/ofid/ofad171