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Study · Non-randomized trial · n = 78 · 2021

6 results

A phase 2 trial of the somatostatin analog pasireotide to prevent GI toxicity and acute GVHD in allogeneic hematopoietic stem cell transplant.

Ramalingam S, Siamakpour-Reihani S, Bohannan L, Ren Y, Sibley A, Sheng J, Ma L, Nixon AB, Lyu J, Parker DC, Bain J, Muehlbauer M and 17 more

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Ilkayeva O, Kraus VB, Huebner JL, Spitzer T, Brown J, Peled JU, van den Brink M, Gomes A, Choi T, Gasparetto C, Horwitz M, Long G, Lopez R, Rizzieri D, Sarantopoulos S, Chao N, Sung AD

PloS one25 Jun 2021Cited by 3 (Europe PMC)

PMID34170918 (opens in a new tab)PMCIDPMC8232534 (opens in a new tab)DOI10.1371/journal.pone.0252995 (opens in a new tab)PepBankPUB-34170918
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Summary

BackgroundAllogeneic hematopoietic stem cell transplantation (HCT) is an often curative intent treatment, however it is associated with significant gastrointestinal (GI) toxicity and treatment related mortality. Graft-versus-host disease is a significant contributor to transplant-related mortality. We performed a phase 2 trial of the somatostatin analog pasireotide to prevent gastrointestinal toxicity and GVHD after myeloablative allogeneic HCT.MethodsPatients received 0.9mg pasireotide every 12 hours from the day prior to conditioning through day +4 after HCT (or a maximum of 14 days). The primary outcomes were grade 3-4 gastrointestinal toxicity through day 30 and acute GVHD. Secondary outcomes were chronic GVHD, overall survival and relapse free survival at one year. Stool and blood samples were collected from before and after HCT for analyses of stool microbiome, local inflammatory markers, and systemic inflammatory and metabolic markers. Results were compared with matched controls.ResultsTwenty-six patients received pasireotide and were compared to 52 matched contemporaneous controls using a 1-2 match. Grade 3-4 GI toxicity occurred in 21 (81%) patients who received pasireotide and 35 (67%) controls (p = 0.33). Acute GVHD occurred in 15 (58%) patients in the pasireotide group and 28 (54%) controls (p = 0.94). Chronic GVHD occurred in 16 patients in the pasireotide group (64%) versus 22 patients in the control group (42%) (p = 0.12). Overall survival at 1 year in the pasireotide group was 63% (95% CI: 47%,86%) versus 82% (95% CI: 72%, 93%) in controls (log-rank p = 0.006). Relapse-free survival rate at one year was 40% (95% CI: 25%, 65%) in the pasireotide group versus 78% (95% CI: 68%, 91%) in controls (log-rank p = 0.002). After controlling for the effect of relevant covariates, patients in the pasireotide group had attenuated post-HCT loss of microbial diversity. Analysis of systemic inflammatory markers and metabolomics demonstrated feasibility of such analyses in patients undergoing allogeneic HCT. Baseline level and pre-to-post transplant changes in several inflammatory markers (including MIP1a, MIP1b, TNFa, IL8Pro, and IL6) correlated with likelihood of survival.ConclusionsPasireotide did not prevent gastrointestinal toxicity or acute GVHD compared to contemporaneous controls. Pasireotide was associated with numerically higher chronic GVHD and significantly decreased OS and RFS compared to contemporaneous controls. Pasireotide may provide a locally protective effect in the stool microbiome and in local inflammation as measured by stool calprotectin, stool beta-defensin, and stool diversity index.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study6

6 results from studies that gave the peptide. 6 results from people.

  1. 01HumanNon-randomized trialSafety outcome

    After allogeneic HCT, grade 3-4 GI toxicity occurred in 21 (81%) pasireotide patients versus 35 (67%) controls (p = 0.33).

    In plain wordsAfter allogeneic HCT, severe gastrointestinal toxicity was numerically higher with pasireotide than matched controls, without a significant difference.

    Peptide
    Pasireotide
    Outcome
    IncreaseGrade 3-4 gastrointestinal toxicity through day 30
    Study

    n = 78

    Comparator
    Matched contemporaneous controls
    Analysis
    Primary
    Outcome group
    Gastrointestinal adverse events · Gastrointestinal
    Studied dose
    0.9mg every 12 hours · day prior to conditioning through day +4 after HCT, maximum 14 daysAs given in this study (human).

    Measurements

    Grade 3-4 GI toxicity, patients
    21 patients
    pasireotide group
    Grade 3-4 GI toxicity
    81%
    pasireotide group
    Grade 3-4 GI toxicity, patients
    35 patients
    matched contemporaneous controls
    Grade 3-4 GI toxicity
    67%
    matched contemporaneous controls
    p value
    0.33 p-value
    pasireotide vs matched contemporaneous controls

    Source sentences

    Summary
    Grade 3-4 GI toxicity occurred in 21 (81%) patients who received pasireotide and 35 (67%) controls (p = 0.33).
    4 context sentences
    Summary · context
    The primary outcomes were grade 3-4 gastrointestinal toxicity through day 30 and acute GVHD.
    Summary · context
    We performed a phase 2 trial of the somatostatin analog pasireotide to prevent gastrointestinal toxicity and GVHD after myeloablative allogeneic HCT.
    Summary · context
    Patients received 0.9mg pasireotide every 12 hours from the day prior to conditioning through day +4 after HCT (or a maximum of 14 days).
    Summary · context
    Twenty-six patients received pasireotide and were compared to 52 matched contemporaneous controls using a 1-2 match.
  2. 02HumanNon-randomized trialEfficacy outcome

    After allogeneic HCT, acute GVHD occurred in 15 (58%) pasireotide patients versus 28 (54%) controls (p = 0.94).

    In plain wordsAfter allogeneic HCT, acute GVHD was numerically higher with pasireotide than matched controls, without a significant difference.

    Peptide
    Pasireotide
    Outcome
    IncreaseAcute GVHD
    Study

    n = 78

    Comparator
    Matched contemporaneous controls
    Analysis
    Primary
    Outcome group
    Immune response · Immune and inflammation
    Studied dose
    0.9mg every 12 hours · day prior to conditioning through day +4 after HCT, maximum 14 daysAs given in this study (human).

    Measurements

    Acute GVHD, patients
    15 patients
    pasireotide group
    Acute GVHD
    58%
    pasireotide group
    Acute GVHD, patients
    28 patients
    matched contemporaneous controls
    Acute GVHD
    54%
    matched contemporaneous controls
    p value
    0.94 p-value
    pasireotide vs matched contemporaneous controls

    Source sentences

    Summary
    Acute GVHD occurred in 15 (58%) patients in the pasireotide group and 28 (54%) controls (p = 0.94).
    3 context sentences
    Summary · context
    We performed a phase 2 trial of the somatostatin analog pasireotide to prevent gastrointestinal toxicity and GVHD after myeloablative allogeneic HCT.
    Summary · context
    Patients received 0.9mg pasireotide every 12 hours from the day prior to conditioning through day +4 after HCT (or a maximum of 14 days).
    Summary · context
    Twenty-six patients received pasireotide and were compared to 52 matched contemporaneous controls using a 1-2 match.
  3. 03HumanNon-randomized trialEfficacy outcome

    After allogeneic HCT, chronic GVHD occurred in 16 (64%) pasireotide patients versus 22 (42%) controls (p = 0.12).

    In plain wordsAfter allogeneic HCT, chronic GVHD was numerically higher with pasireotide than matched controls, without a significant difference.

    Peptide
    Pasireotide
    Outcome
    IncreaseChronic GVHD
    Study

    n: not reported in source

    Comparator
    Matched contemporaneous controls
    Analysis
    Secondary
    Outcome group
    Immune response · Immune and inflammation
    Studied dose
    0.9mg every 12 hours · day prior to conditioning through day +4 after HCT, maximum 14 daysAs given in this study (human).

    Measurements

    Chronic GVHD, patients
    16 patients
    pasireotide group
    Chronic GVHD
    64%
    pasireotide group
    Chronic GVHD, patients
    22 patients
    control group
    Chronic GVHD
    42%
    control group
    p value
    0.12 p-value
    pasireotide vs control

    Source sentences

    Summary
    Chronic GVHD occurred in 16 patients in the pasireotide group (64%) versus 22 patients in the control group (42%) (p = 0.12).
    3 context sentences
    Summary · context
    We performed a phase 2 trial of the somatostatin analog pasireotide to prevent gastrointestinal toxicity and GVHD after myeloablative allogeneic HCT.
    Summary · context
    Patients received 0.9mg pasireotide every 12 hours from the day prior to conditioning through day +4 after HCT (or a maximum of 14 days).
    Summary · context
    Twenty-six patients received pasireotide and were compared to 52 matched contemporaneous controls using a 1-2 match.
  4. 04HumanNon-randomized trialEfficacy outcome

    After allogeneic HCT, one-year survival was 63% (95% CI 47%,86%) with pasireotide versus 82% (72%,93%) in controls (p = 0.006).

    In plain wordsAfter allogeneic HCT, one-year overall survival was lower with pasireotide than matched controls.

    Peptide
    Pasireotide
    Outcome
    DecreaseOne-year overall survival
    Study

    n: not reported in source

    Comparator
    Matched contemporaneous controls
    Analysis
    Secondary
    Outcome group
    Mortality · Whole body
    Studied dose
    0.9mg every 12 hours · day prior to conditioning through day +4 after HCT, maximum 14 daysAs given in this study (human).

    Measurements

    Overall survival at 1 year
    63%
    pasireotide group
    95% CI, overall survival
    47 to 86%
    pasireotide group
    Overall survival at 1 year
    82%
    controls
    95% CI, overall survival
    72 to 93%
    controls
    Log-rank p value
    0.006 p-value
    pasireotide vs controls

    Source sentences

    Summary
    Overall survival at 1 year in the pasireotide group was 63% (95% CI: 47%,86%) versus 82% (95% CI: 72%, 93%) in controls (log-rank p = 0.006).
    3 context sentences
    Summary · context
    We performed a phase 2 trial of the somatostatin analog pasireotide to prevent gastrointestinal toxicity and GVHD after myeloablative allogeneic HCT.
    Summary · context
    Patients received 0.9mg pasireotide every 12 hours from the day prior to conditioning through day +4 after HCT (or a maximum of 14 days).
    Summary · context
    Twenty-six patients received pasireotide and were compared to 52 matched contemporaneous controls using a 1-2 match.
  5. 05HumanNon-randomized trialEfficacy outcome

    After allogeneic HCT, one-year relapse-free survival was 40% (95% CI 25%,65%) with pasireotide versus 78% (68%,91%) in controls (p = 0.002).

    In plain wordsAfter allogeneic HCT, one-year relapse-free survival was lower with pasireotide than matched controls.

    Peptide
    Pasireotide
    Outcome
    DecreaseOne-year relapse-free survival
    Study

    n: not reported in source

    Comparator
    Matched contemporaneous controls
    Analysis
    Secondary
    Outcome group
    Tumor growth · Cancer
    Studied dose
    0.9mg every 12 hours · day prior to conditioning through day +4 after HCT, maximum 14 daysAs given in this study (human).

    Measurements

    Relapse-free survival at 1 year
    40%
    pasireotide group
    95% CI, relapse-free survival
    25 to 65%
    pasireotide group
    Relapse-free survival at 1 year
    78%
    controls
    95% CI, relapse-free survival
    68 to 91%
    controls
    Log-rank p value
    0.002 p-value
    pasireotide vs controls

    Source sentences

    Summary
    Relapse-free survival rate at one year was 40% (95% CI: 25%, 65%) in the pasireotide group versus 78% (95% CI: 68%, 91%) in controls (log-rank p = 0.002).
    3 context sentences
    Summary · context
    We performed a phase 2 trial of the somatostatin analog pasireotide to prevent gastrointestinal toxicity and GVHD after myeloablative allogeneic HCT.
    Summary · context
    Patients received 0.9mg pasireotide every 12 hours from the day prior to conditioning through day +4 after HCT (or a maximum of 14 days).
    Summary · context
    Twenty-six patients received pasireotide and were compared to 52 matched contemporaneous controls using a 1-2 match.
  6. 06HumanObservational studyMechanism

    After adjustment for relevant covariates, pasireotide exposure was associated with attenuated post-HCT loss of microbial diversity versus matched controls.

    In plain wordsAfter allogeneic HCT, pasireotide exposure was linked to less loss of microbial diversity than matched controls after adjustment.

    Peptide
    Pasireotide
    Outcome
    DecreasePost-HCT loss of microbial diversity
    Study

    n: not reported in source

    Comparator
    Matched contemporaneous controls
    Outcome group
    Gut microbiota · Gastrointestinal
    Studied dose
    0.9mg every 12 hours · day prior to conditioning through day +4 after HCT, maximum 14 daysAs given in this study (human).

    Source sentences

    Summary
    After controlling for the effect of relevant covariates, patients in the pasireotide group had attenuated post-HCT loss of microbial diversity.
    3 context sentences
    Summary · context
    We performed a phase 2 trial of the somatostatin analog pasireotide to prevent gastrointestinal toxicity and GVHD after myeloablative allogeneic HCT.
    Summary · context
    Patients received 0.9mg pasireotide every 12 hours from the day prior to conditioning through day +4 after HCT (or a maximum of 14 days).
    Summary · context
    Twenty-six patients received pasireotide and were compared to 52 matched contemporaneous controls using a 1-2 match.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:34 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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