EFFICACY AND SAFETY OF LENALIDOMIDE AND DEXAMETHASONE COMBINATION IN PATIENTS WITH POEMS SYNDROME PRE-TREATED OR INELIGIBLE FOR HIGH-DOSE THERAPY: RESULTS OF A PROSPECTIVE TRIAL.
(Abstract release date: 05/21/15)
EHA Library. Nozza A. 06/12/15; 101349; LB2086
Disclosure(s): Humanitas Cancer CenterHematology
Andrea Nozza
Contributions
Contributions
Abstract
Abstract: LB2086
Type: Eposter Presentation
Background
Aims
This study plans to evaluate efficacy and toxicity of Lenalidomide in POEMS.
Lenalidomide 25 mg/day was given for 21 days in association with weekly Dexamethasone 40 mg until progression or toxicity.
Response to treatment was assessed by a specifically prepared clinical scale which evaluates ten hematological and clinical parameters( clinical response evaluation scale ', CRES, range 0-20), as welll as by the Overall Neuropathy Limitations Scale (ONLS), an expanded MRC sum score, the INCAT sensory sum score and EMG, to evaluate neurological response. The primary end-point was the response to therapy after six monthly cycles of RD. Pts were considered responders if they showed an improvement of at least 1 point in the ONLS scale and at least 3 in the CRES scale
Methods
From 10/09 to 10/14, we performed a pilot study with Lenalidomide plus dexamethasone (RD) in 20 patients with POEMS - All Pts had a median ONLS score of 4.5 (range 1-8), and monoclonal component (lambda restricted in 88%) - Serum VEGF levels were >1000 pg/ml in all but two pts (88.9%) with a median level of 4043 pg/ml (range 687-13856). Osteoscleoriotic bone lesions were found by X-ray in 12 pts (67%), while Castelman Disease was histologicallly confirmed in 2 pts. Skin changes were observed in 16 pts (89%), hepatosplenomegaly in 14 (78%) pleural effusion, ascites or peripheral edema in 6 pts (33 %) and endocrine alterations in 16 (89%)
Results
Response evaluation after 6 months of RD was done in 16 out of 18 pts: 2 pts had dropped out during the first cycle (1 lost to follow-up, 1 for withdrawal of informed consent): 15/18 of included pts (83.3%) completed the 6 cycles of therapy and were improved at six month in the clinical or neurological scale or both, while one patient not improved and suspended the treatment after 4 cycles. 13 pts (72.2%) had improved by at least 3 points in CRES, ranging from 3 to 7 points (mean score at entry 10.36; mean score at six month 7.18; p= 0.0006) . 10 pts (55.6%) improved by at least one point in the ONLS with an improvement ranging from 1 to 3 points (mean score at entry 4.3; mean score at six month 3.6; p = 0.0176), five remained stable and one patient had deteriorated by two points.
Six patients are still on treatment, after a median follow-up of 33 months (range 10-52 months)- 5 Pts discontinued treatment for complete response-stable disease after a median of 30 months (range 25-32 months) and only one relapsed 20 months later. In 4 pts the disease progressed after 10, 21, 29 and 35 months. No patient discontinued treatment due to toxicity. Lenalidomide was reduced in 43% of patients mainly for haematological toxicity (grade II-III), and dexamentasone was reduced in all patients, and stopped in 30%. Has not been reported any thrombotic events or secondary neoplasia in patients enrolled.
Summary
Conclusions: This prospective trail confirms that lenalidomide is very effective and well tolerated in the majority of patients, regardless of any previous treatments and it has a prolonged efficacy in POEMS Syndrome
Keyword(s): VEGF
Session topic: E-poster
Type: Eposter Presentation
Background
Introduction: POEMS syndrome is a rare multisystemic disease. Several treatments have been proposed for POEMS even if there is so far no controlled study. Lenalidomide has anti-angiogenic activity through inhibition of VEGF and TNF alpha.
Aims
This study plans to evaluate efficacy and toxicity of Lenalidomide in POEMS.
Lenalidomide 25 mg/day was given for 21 days in association with weekly Dexamethasone 40 mg until progression or toxicity.
Response to treatment was assessed by a specifically prepared clinical scale which evaluates ten hematological and clinical parameters( clinical response evaluation scale ', CRES, range 0-20), as welll as by the Overall Neuropathy Limitations Scale (ONLS), an expanded MRC sum score, the INCAT sensory sum score and EMG, to evaluate neurological response. The primary end-point was the response to therapy after six monthly cycles of RD. Pts were considered responders if they showed an improvement of at least 1 point in the ONLS scale and at least 3 in the CRES scale
Methods
From 10/09 to 10/14, we performed a pilot study with Lenalidomide plus dexamethasone (RD) in 20 patients with POEMS - All Pts had a median ONLS score of 4.5 (range 1-8), and monoclonal component (lambda restricted in 88%) - Serum VEGF levels were >1000 pg/ml in all but two pts (88.9%) with a median level of 4043 pg/ml (range 687-13856). Osteoscleoriotic bone lesions were found by X-ray in 12 pts (67%), while Castelman Disease was histologicallly confirmed in 2 pts. Skin changes were observed in 16 pts (89%), hepatosplenomegaly in 14 (78%) pleural effusion, ascites or peripheral edema in 6 pts (33 %) and endocrine alterations in 16 (89%)
Results
Response evaluation after 6 months of RD was done in 16 out of 18 pts: 2 pts had dropped out during the first cycle (1 lost to follow-up, 1 for withdrawal of informed consent): 15/18 of included pts (83.3%) completed the 6 cycles of therapy and were improved at six month in the clinical or neurological scale or both, while one patient not improved and suspended the treatment after 4 cycles. 13 pts (72.2%) had improved by at least 3 points in CRES, ranging from 3 to 7 points (mean score at entry 10.36; mean score at six month 7.18; p= 0.0006) . 10 pts (55.6%) improved by at least one point in the ONLS with an improvement ranging from 1 to 3 points (mean score at entry 4.3; mean score at six month 3.6; p = 0.0176), five remained stable and one patient had deteriorated by two points.
Six patients are still on treatment, after a median follow-up of 33 months (range 10-52 months)- 5 Pts discontinued treatment for complete response-stable disease after a median of 30 months (range 25-32 months) and only one relapsed 20 months later. In 4 pts the disease progressed after 10, 21, 29 and 35 months. No patient discontinued treatment due to toxicity. Lenalidomide was reduced in 43% of patients mainly for haematological toxicity (grade II-III), and dexamentasone was reduced in all patients, and stopped in 30%. Has not been reported any thrombotic events or secondary neoplasia in patients enrolled.
Summary
Conclusions: This prospective trail confirms that lenalidomide is very effective and well tolerated in the majority of patients, regardless of any previous treatments and it has a prolonged efficacy in POEMS Syndrome
Keyword(s): VEGF
Session topic: E-poster
Abstract: LB2086
Type: Eposter Presentation
Background
Aims
This study plans to evaluate efficacy and toxicity of Lenalidomide in POEMS.
Lenalidomide 25 mg/day was given for 21 days in association with weekly Dexamethasone 40 mg until progression or toxicity.
Response to treatment was assessed by a specifically prepared clinical scale which evaluates ten hematological and clinical parameters( clinical response evaluation scale ', CRES, range 0-20), as welll as by the Overall Neuropathy Limitations Scale (ONLS), an expanded MRC sum score, the INCAT sensory sum score and EMG, to evaluate neurological response. The primary end-point was the response to therapy after six monthly cycles of RD. Pts were considered responders if they showed an improvement of at least 1 point in the ONLS scale and at least 3 in the CRES scale
Methods
From 10/09 to 10/14, we performed a pilot study with Lenalidomide plus dexamethasone (RD) in 20 patients with POEMS - All Pts had a median ONLS score of 4.5 (range 1-8), and monoclonal component (lambda restricted in 88%) - Serum VEGF levels were >1000 pg/ml in all but two pts (88.9%) with a median level of 4043 pg/ml (range 687-13856). Osteoscleoriotic bone lesions were found by X-ray in 12 pts (67%), while Castelman Disease was histologicallly confirmed in 2 pts. Skin changes were observed in 16 pts (89%), hepatosplenomegaly in 14 (78%) pleural effusion, ascites or peripheral edema in 6 pts (33 %) and endocrine alterations in 16 (89%)
Results
Response evaluation after 6 months of RD was done in 16 out of 18 pts: 2 pts had dropped out during the first cycle (1 lost to follow-up, 1 for withdrawal of informed consent): 15/18 of included pts (83.3%) completed the 6 cycles of therapy and were improved at six month in the clinical or neurological scale or both, while one patient not improved and suspended the treatment after 4 cycles. 13 pts (72.2%) had improved by at least 3 points in CRES, ranging from 3 to 7 points (mean score at entry 10.36; mean score at six month 7.18; p= 0.0006) . 10 pts (55.6%) improved by at least one point in the ONLS with an improvement ranging from 1 to 3 points (mean score at entry 4.3; mean score at six month 3.6; p = 0.0176), five remained stable and one patient had deteriorated by two points.
Six patients are still on treatment, after a median follow-up of 33 months (range 10-52 months)- 5 Pts discontinued treatment for complete response-stable disease after a median of 30 months (range 25-32 months) and only one relapsed 20 months later. In 4 pts the disease progressed after 10, 21, 29 and 35 months. No patient discontinued treatment due to toxicity. Lenalidomide was reduced in 43% of patients mainly for haematological toxicity (grade II-III), and dexamentasone was reduced in all patients, and stopped in 30%. Has not been reported any thrombotic events or secondary neoplasia in patients enrolled.
Summary
Conclusions: This prospective trail confirms that lenalidomide is very effective and well tolerated in the majority of patients, regardless of any previous treatments and it has a prolonged efficacy in POEMS Syndrome
Keyword(s): VEGF
Session topic: E-poster
Type: Eposter Presentation
Background
Introduction: POEMS syndrome is a rare multisystemic disease. Several treatments have been proposed for POEMS even if there is so far no controlled study. Lenalidomide has anti-angiogenic activity through inhibition of VEGF and TNF alpha.
Aims
This study plans to evaluate efficacy and toxicity of Lenalidomide in POEMS.
Lenalidomide 25 mg/day was given for 21 days in association with weekly Dexamethasone 40 mg until progression or toxicity.
Response to treatment was assessed by a specifically prepared clinical scale which evaluates ten hematological and clinical parameters( clinical response evaluation scale ', CRES, range 0-20), as welll as by the Overall Neuropathy Limitations Scale (ONLS), an expanded MRC sum score, the INCAT sensory sum score and EMG, to evaluate neurological response. The primary end-point was the response to therapy after six monthly cycles of RD. Pts were considered responders if they showed an improvement of at least 1 point in the ONLS scale and at least 3 in the CRES scale
Methods
From 10/09 to 10/14, we performed a pilot study with Lenalidomide plus dexamethasone (RD) in 20 patients with POEMS - All Pts had a median ONLS score of 4.5 (range 1-8), and monoclonal component (lambda restricted in 88%) - Serum VEGF levels were >1000 pg/ml in all but two pts (88.9%) with a median level of 4043 pg/ml (range 687-13856). Osteoscleoriotic bone lesions were found by X-ray in 12 pts (67%), while Castelman Disease was histologicallly confirmed in 2 pts. Skin changes were observed in 16 pts (89%), hepatosplenomegaly in 14 (78%) pleural effusion, ascites or peripheral edema in 6 pts (33 %) and endocrine alterations in 16 (89%)
Results
Response evaluation after 6 months of RD was done in 16 out of 18 pts: 2 pts had dropped out during the first cycle (1 lost to follow-up, 1 for withdrawal of informed consent): 15/18 of included pts (83.3%) completed the 6 cycles of therapy and were improved at six month in the clinical or neurological scale or both, while one patient not improved and suspended the treatment after 4 cycles. 13 pts (72.2%) had improved by at least 3 points in CRES, ranging from 3 to 7 points (mean score at entry 10.36; mean score at six month 7.18; p= 0.0006) . 10 pts (55.6%) improved by at least one point in the ONLS with an improvement ranging from 1 to 3 points (mean score at entry 4.3; mean score at six month 3.6; p = 0.0176), five remained stable and one patient had deteriorated by two points.
Six patients are still on treatment, after a median follow-up of 33 months (range 10-52 months)- 5 Pts discontinued treatment for complete response-stable disease after a median of 30 months (range 25-32 months) and only one relapsed 20 months later. In 4 pts the disease progressed after 10, 21, 29 and 35 months. No patient discontinued treatment due to toxicity. Lenalidomide was reduced in 43% of patients mainly for haematological toxicity (grade II-III), and dexamentasone was reduced in all patients, and stopped in 30%. Has not been reported any thrombotic events or secondary neoplasia in patients enrolled.
Summary
Conclusions: This prospective trail confirms that lenalidomide is very effective and well tolerated in the majority of patients, regardless of any previous treatments and it has a prolonged efficacy in POEMS Syndrome
Keyword(s): VEGF
Session topic: E-poster
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