Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma): A single-center experience

Anita D'Souza, Martha Lacy, Morie Gertz, Shaji K Kumar, Francis Buadi, Suzanne Hayman, David M Dingli, Steven Zeldenrust, Robert Kyle, Stephen Maxted Ansell, David Inwards, Patrick Bruce Johnston, Ivana Micallef, Luis Porrata, Mark R Litzow, Dennis Gastineau, William Hogan, Angela Dispenzieri

Research output: Contribution to journalArticle

79 Citations (Scopus)

Abstract

The POEMS syndrome (polyradiculoneuropathy, organomegaly, multiple endocrinopathies, monoclonal protein, skin changes) is a rare disease associated with a plasma cell dyscrasia. Patients with disseminated POEMS can be treated with high-dose therapy and autologous stem cell transplantation (ASCT). While clinical improvement is nearly universal in these patients, the long-term outcomes after transplantation are unclear.We therefore assessed the long-term clinical outcomes of 59 POEMS patients treated with ASCT at our institution. With a median follow-up of 45 months, 14 patients have progressed with a progression-free survival of 98% and 75% at 1 and 5 years, respectively. Factors associated with progression have included an IgG-λ monoclonal component (hazard ratio [HR] 7.5; 95% confidence interval [CI], 2.3-28.3; P = .0008), fluorodeoxyglucose-avid lesions on baseline positron emission tomography (HR 6.4; 95% CI, 1.2-120; P = .03), lack of complete hematologic response (HR 5.4; 95% CI, 1.8-16.7; P = .003), and patients aged 50 years or younger at transplantation (HR 4.4; 95% CI, 1.3-20; P = .01). The most common progression events have been radiologic followed by rising VEGF. Symptomatic progression has been rare. Most patients could be salvaged with immunomodulatory drugs or radiation. The 5-year survival is 94%. Herein, we describe a system of monitoring response and progression among patients with POEMS after ASCT.

Original languageEnglish (US)
Pages (from-to)56-62
Number of pages7
JournalBlood
Volume120
Issue number1
DOIs
StatePublished - Jul 5 2012

Fingerprint

POEMS Syndrome
Stem Cell Transplantation
Stem cells
Hazards
Confidence Intervals
Positron emission tomography
Vascular Endothelial Growth Factor A
Transplantation
Polyradiculoneuropathy
Skin
Immunoglobulin G
Paraproteinemias
Plasmas
Radiation
Rare Diseases
Monitoring
Positron-Emission Tomography
Disease-Free Survival
Pharmaceutical Preparations
Proteins

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma) : A single-center experience. / D'Souza, Anita; Lacy, Martha; Gertz, Morie; Kumar, Shaji K; Buadi, Francis; Hayman, Suzanne; Dingli, David M; Zeldenrust, Steven; Kyle, Robert; Ansell, Stephen Maxted; Inwards, David; Johnston, Patrick Bruce; Micallef, Ivana; Porrata, Luis; Litzow, Mark R; Gastineau, Dennis; Hogan, William; Dispenzieri, Angela.

In: Blood, Vol. 120, No. 1, 05.07.2012, p. 56-62.

Research output: Contribution to journalArticle

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abstract = "The POEMS syndrome (polyradiculoneuropathy, organomegaly, multiple endocrinopathies, monoclonal protein, skin changes) is a rare disease associated with a plasma cell dyscrasia. Patients with disseminated POEMS can be treated with high-dose therapy and autologous stem cell transplantation (ASCT). While clinical improvement is nearly universal in these patients, the long-term outcomes after transplantation are unclear.We therefore assessed the long-term clinical outcomes of 59 POEMS patients treated with ASCT at our institution. With a median follow-up of 45 months, 14 patients have progressed with a progression-free survival of 98{\%} and 75{\%} at 1 and 5 years, respectively. Factors associated with progression have included an IgG-λ monoclonal component (hazard ratio [HR] 7.5; 95{\%} confidence interval [CI], 2.3-28.3; P = .0008), fluorodeoxyglucose-avid lesions on baseline positron emission tomography (HR 6.4; 95{\%} CI, 1.2-120; P = .03), lack of complete hematologic response (HR 5.4; 95{\%} CI, 1.8-16.7; P = .003), and patients aged 50 years or younger at transplantation (HR 4.4; 95{\%} CI, 1.3-20; P = .01). The most common progression events have been radiologic followed by rising VEGF. Symptomatic progression has been rare. Most patients could be salvaged with immunomodulatory drugs or radiation. The 5-year survival is 94{\%}. Herein, we describe a system of monitoring response and progression among patients with POEMS after ASCT.",
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T1 - Long-term outcomes after autologous stem cell transplantation for patients with POEMS syndrome (osteosclerotic myeloma)

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AU - D'Souza, Anita

AU - Lacy, Martha

AU - Gertz, Morie

AU - Kumar, Shaji K

AU - Buadi, Francis

AU - Hayman, Suzanne

AU - Dingli, David M

AU - Zeldenrust, Steven

AU - Kyle, Robert

AU - Ansell, Stephen Maxted

AU - Inwards, David

AU - Johnston, Patrick Bruce

AU - Micallef, Ivana

AU - Porrata, Luis

AU - Litzow, Mark R

AU - Gastineau, Dennis

AU - Hogan, William

AU - Dispenzieri, Angela

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N2 - The POEMS syndrome (polyradiculoneuropathy, organomegaly, multiple endocrinopathies, monoclonal protein, skin changes) is a rare disease associated with a plasma cell dyscrasia. Patients with disseminated POEMS can be treated with high-dose therapy and autologous stem cell transplantation (ASCT). While clinical improvement is nearly universal in these patients, the long-term outcomes after transplantation are unclear.We therefore assessed the long-term clinical outcomes of 59 POEMS patients treated with ASCT at our institution. With a median follow-up of 45 months, 14 patients have progressed with a progression-free survival of 98% and 75% at 1 and 5 years, respectively. Factors associated with progression have included an IgG-λ monoclonal component (hazard ratio [HR] 7.5; 95% confidence interval [CI], 2.3-28.3; P = .0008), fluorodeoxyglucose-avid lesions on baseline positron emission tomography (HR 6.4; 95% CI, 1.2-120; P = .03), lack of complete hematologic response (HR 5.4; 95% CI, 1.8-16.7; P = .003), and patients aged 50 years or younger at transplantation (HR 4.4; 95% CI, 1.3-20; P = .01). The most common progression events have been radiologic followed by rising VEGF. Symptomatic progression has been rare. Most patients could be salvaged with immunomodulatory drugs or radiation. The 5-year survival is 94%. Herein, we describe a system of monitoring response and progression among patients with POEMS after ASCT.

AB - The POEMS syndrome (polyradiculoneuropathy, organomegaly, multiple endocrinopathies, monoclonal protein, skin changes) is a rare disease associated with a plasma cell dyscrasia. Patients with disseminated POEMS can be treated with high-dose therapy and autologous stem cell transplantation (ASCT). While clinical improvement is nearly universal in these patients, the long-term outcomes after transplantation are unclear.We therefore assessed the long-term clinical outcomes of 59 POEMS patients treated with ASCT at our institution. With a median follow-up of 45 months, 14 patients have progressed with a progression-free survival of 98% and 75% at 1 and 5 years, respectively. Factors associated with progression have included an IgG-λ monoclonal component (hazard ratio [HR] 7.5; 95% confidence interval [CI], 2.3-28.3; P = .0008), fluorodeoxyglucose-avid lesions on baseline positron emission tomography (HR 6.4; 95% CI, 1.2-120; P = .03), lack of complete hematologic response (HR 5.4; 95% CI, 1.8-16.7; P = .003), and patients aged 50 years or younger at transplantation (HR 4.4; 95% CI, 1.3-20; P = .01). The most common progression events have been radiologic followed by rising VEGF. Symptomatic progression has been rare. Most patients could be salvaged with immunomodulatory drugs or radiation. The 5-year survival is 94%. Herein, we describe a system of monitoring response and progression among patients with POEMS after ASCT.

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