Trial Outcomes & Findings for Bronchodilator Responsiveness in Obliterative Bronchiolitis (NCT NCT01112241)

NCT ID: NCT01112241

Last Updated: 2011-03-02

Results Overview

Following albuterol plus tiotropium inhalation, FEV1 increments ≥12 per cent as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to the American Thoracic Society-European Respiratory Society standard criteria \[Pellegrino et al. Eur Respir J 2005; 26: 948-968\]. They were calculated as follows: \[FEV1, expressed in liters (L), after bronchodilators - FEV1 (L) before bronchodilators/FEV1 (L) before bronchodilators x 100\].

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

17 participants

Primary outcome timeframe

Baseline and 90 min after bronchodilators

Results posted on

2011-03-02

Participant Flow

Between April 2010 and May 2010, seventeen consecutive bronchiolitis obliterans (BOS) patients following hematopoietic stem cell transplantation (HSCT) were studied.

In all patients, airflow obstruction was found at spirometry between 14 and 72 months (median, 41 months) after HSCT. The diagnosis of BOS was confirmed by fulfilment of currently updated criteria \[Filipovich AH et al. Biol Blood Marrow Transplant 2005; 11:945-956\]\[Chien JW et al. Biol Blood Marrow Transplant 2010; 16:S106-S114\].

Participant milestones

Participant milestones
Measure
Albuterol Plus Tiotropium
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Overall Study
STARTED
17
Overall Study
COMPLETED
17
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Bronchodilator Responsiveness in Obliterative Bronchiolitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
17 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age Continuous
48 years
STANDARD_DEVIATION 12 • n=5 Participants
Sex: Female, Male
Female
5 Participants
n=5 Participants
Sex: Female, Male
Male
12 Participants
n=5 Participants
Region of Enrollment
Italy
17 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, FEV1 increments ≥12 per cent as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to the American Thoracic Society-European Respiratory Society standard criteria \[Pellegrino et al. Eur Respir J 2005; 26: 948-968\]. They were calculated as follows: \[FEV1, expressed in liters (L), after bronchodilators - FEV1 (L) before bronchodilators/FEV1 (L) before bronchodilators x 100\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Per Cent Change of Forced Expiratory Volume in 1 Second (FEV1) After Bronchodilators
Responsives to bronchodilators (n=4)
14 per cent positive change
Standard Deviation 1
Per Cent Change of Forced Expiratory Volume in 1 Second (FEV1) After Bronchodilators
Unresponsives to bronchodilators (n=13)
7 per cent positive change
Standard Deviation 5

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, FEV1 increments ≥0.20 liters (L) as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to the American Thoracic Society-European Respiratory Society standard criteria \[Pellegrino et al. Eur Respir J 2005; 26: 948-968\]. They were calculated as follows: \[FEV1 (L) after bronchodilators - FEV1 (L) before bronchodilators\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Absolute Change of Forced Expiratory Volume in 1 Second (FEV1) After Bronchodilators
Responsives to bronchodilators (n=4)
0.26 absolute positive change (liters)
Standard Deviation 0.06
Absolute Change of Forced Expiratory Volume in 1 Second (FEV1) After Bronchodilators
Unresponsives to bronchodilators (n=13)
0.12 absolute positive change (liters)
Standard Deviation 0.09

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, FVC increments ≥12 per cent as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to the American Thoracic Society-European Respiratory Society standard criteria \[Pellegrino et al. Eur Respir J 2005; 26: 948-968\]. They were calculated as follows: \[FVC, expressed in liters (L), after bronchodilators - FVC (L) before bronchodilators/FVC (L) before bronchodilators x 100\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Per Cent Change of Forced Vital Capacity (FVC) After Bronchodilators
Responsives to bronchodilators (n=5)
24 per cent positive change
Standard Deviation 13
Per Cent Change of Forced Vital Capacity (FVC) After Bronchodilators
Unresponsives to bronchodilators (n=12)
2 per cent positive change
Standard Deviation 4

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, FVC increments ≥0.20 liters (L) compared with baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to the American Thoracic Society-European Respiratory Society standard criteria \[Pellegrino et al. Eur Respir J 2005; 26: 948-968\]. They were calculated as follows: \[FVC (L) after bronchodilators - FVC (L) before bronchodilators\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Absolute Change of Forced Vital Capacity (FVC) After Bronchodilators
Responsives to bronchodilators (n=5)
0.65 absolute positive change (liters)
Standard Deviation 0.37
Absolute Change of Forced Vital Capacity (FVC) After Bronchodilators
Unresponsives to bronchodilators (n=12)
0.07 absolute positive change (liters)
Standard Deviation 0.15

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following HSCT for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, V'max increments ≥40 per cent as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to Pellegrino et al. \[Chest 1998; 114:1607-1612\]. They were calculated as follows: \[V'max, expressed in liters.second-1 (L.s-1), after bronchodilators - V'max (L.s-1) before bronchodilators/V'max (L.s-1) before bronchodilators x 100\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Per Cent Change of Instantaneous Maximal Forced Expiratory Flow (V'Max) After Bronchodilators
Responsives to bronchodilators (n=4)
66 per cent positive change
Standard Deviation 17
Per Cent Change of Instantaneous Maximal Forced Expiratory Flow (V'Max) After Bronchodilators
Unresponsives to bronchodilators (n=13)
14 per cent positive change
Standard Deviation 14

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, V'part increments ≥40 per cent as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to Pellegrino et al. \[Chest 1998; 114:1607-1612\]. They were calculated as follows: \[V'part, expressed in liters.second-1 (L.s-1), after bronchodilators - V'part (L.s-1) before bronchodilators/V'part (L.s-1) before bronchodilators x 100\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Per Cent Change of Partial Forced Expiratory Flow (V'Part) After Bronchodilators
Responsives to bronchodilators (n=9)
59 per cent positive change
Standard Deviation 26
Per Cent Change of Partial Forced Expiratory Flow (V'Part) After Bronchodilators
Unresponsives to bronchodilators (n=8)
17 per cent positive change
Standard Deviation 7

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, RV decrements ≥10 per cent as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to O'Donnell et al. \[Eur Respir J 2001; 18: 914-920\]. They were calculated as follows: \[RV, expressed in liters (L), before bronchodilators - RV (L) after bronchodilators/RV (L) after bronchodilators x 100\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Per Cent Change of Residual Volume (RV) After Bronchodilators
Responsives to bronchodilators (n=8)
19 per cent negative change
Standard Deviation 5
Per Cent Change of Residual Volume (RV) After Bronchodilators
Unresponsives to bronchodilators (n=9)
0 per cent negative change
Standard Deviation 5

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, RV decrements ≥0.30 liters (L) as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to O'Donnell et al. \[Eur Respir J 2001; 18: 914-920\]. They were calculated as follows: \[RV (L), before bronchodilators - RV (L) after bronchodilators\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Absolute Change of Residual Volume (RV) After Bronchodilators
Responsives to bronchodilators (n=8)
0.65 absolute negative change (liters)
Standard Deviation 0.37
Absolute Change of Residual Volume (RV) After Bronchodilators
Unresponsives to bronchodilators (n=9)
0.00 absolute negative change (liters)
Standard Deviation 0.10

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, FRC decrements ≥10 per cent as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to O'Donnell et al. \[Eur Respir J 2001; 18: 914-920\]. They were calculated as follows: \[FRC, expressed in liters (L), before bronchodilators - FRC (L) after bronchodilators/FRC (L) after bronchodilators x 100\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Per Cent Change of Functional Residual Capacity (FRC) After Bronchodilators
Responsives to bronchodilators (n=4)
16 per cent negative change
Standard Deviation 6
Per Cent Change of Functional Residual Capacity (FRC) After Bronchodilators
Unresponsives to bronchodilators (n=13)
2 per cent negative change
Standard Deviation 5

PRIMARY outcome

Timeframe: Baseline and 90 min after bronchodilators

Population: Seventeen consecutive, clinically-stable, Caucasian outpatients, presenting with mild-to-very-severe obliterative bronchiolitis following hematopoietic stem cell transplantation (HSCT) for hematological malignancies, were studied. They were diagnosed from 460 patients (253 males, 207 females) undergoing HSCT (sourcing from bone marrow).

Following albuterol plus tiotropium inhalation, FRC decrements ≥0.30 liters (L) as compared to baseline, in an individual subject, were considered as evidence of response to bronchodilators, according to O'Donnell et al. \[Eur Respir J 2001; 18: 914-920\]. They were calculated as follows: \[FRC (L), before bronchodilators - FRC (L) after bronchodilators\].

Outcome measures

Outcome measures
Measure
Albuterol Plus Tiotropium
n=17 Participants
At visit 1, lung function measurements will be performed in triplicate before and 90 min after inhaling four separate doses of 100 μg of albuterol (Ventolin®) and soon after 18 μg of tiotropium bromide \[Spiriva®\] to ensure maximal or near-maximal bronchodilation. Albuterol will be given by a metered-dose inhaler connected to a valved-holding chamber (Volumatic®) and tiotropium by a dry-powder device (Handihaler®).
Absolute Change of Functional Residual Capacity (FRC) After Bronchodilators
Unresponsives to bronchodilators (n=13)
0.07 absolute negative change (liters)
Standard Deviation 0.16
Absolute Change of Functional Residual Capacity (FRC) After Bronchodilators
Responsives to bronchodilators (n=4)
0.78 absolute negative change (liters)
Standard Deviation 0.34

Adverse Events

Albuterol Plus Tiotropium

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Giovanni Barisione, MD

Azienda Ospedaliera Universitaria San Martino, Genova, Italy

Phone: +39 010 5553367

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place