scholarly journals Efficacy and Long-Term Outcomes of Autologous Stem Cell Transplantation in POEMS Syndrome: A Nationwide Survey in Japan

2018 ◽  
Vol 24 (6) ◽  
pp. 1180-1186 ◽  
Author(s):  
Chika Kawajiri-Manako ◽  
Emiko Sakaida ◽  
Chikako Ohwada ◽  
Toshihiro Miyamoto ◽  
Taichi Azuma ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Nationwide Survey ◽  
Poems Syndrome ◽  
Long Term Outcomes

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

Blood ◽  
2012 ◽  
Vol 120 (1) ◽  
pp. 56-62 ◽  
Author(s):  
Anita D'Souza ◽  
Martha Lacy ◽  
Morie Gertz ◽  
Shaji Kumar ◽  
Francis Buadi ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Progression Free Survival ◽  
Poems Syndrome ◽  
Plasma Cell Dyscrasia ◽  
High Dose ◽  
Long Term Outcomes

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.

scholarly journals Long-term outcomes in children with high-risk neuroblastoma treated with autologous stem cell transplantation

2007 ◽  
Vol 40 (8) ◽  
pp. 741-746 ◽  
Author(s):  
T N Trahair ◽  
M R Vowels ◽  
K Johnston ◽  
R J Cohn ◽  
S J Russell ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Long Term Outcomes

scholarly journals Correction: Long-term outcomes of the GPOH NB97 trial for children with high-risk neuroblastoma comparing high-dose chemotherapy with autologous stem cell transplantation and oral chemotherapy as consolidation

2019 ◽  
Vol 121 (10) ◽  
pp. 894-895 ◽  
Author(s):  
Frank Berthold ◽  
Angela Ernst ◽  
Barbara Hero ◽  
Thomas Klingebiel ◽  
Bernhard Kremens ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
High Dose Chemotherapy ◽  
Oral Chemotherapy ◽  
High Dose ◽  
Long Term Outcomes

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Prevalence, clinical profiles, and prognosis of POEMS syndrome in Japanese nationwide survey

Neurology ◽  
2019 ◽  
Vol 93 (10) ◽  
pp. e975-e983 ◽  
Author(s):  
Tomoki Suichi ◽  
Sonoko Misawa ◽  
Minako Beppu ◽  
Sho Takahashi ◽  
Yukari Sekiguchi ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Nationwide Survey ◽  
Poems Syndrome ◽  
Available N ◽  
Skin Changes ◽  
Number Of Patients ◽  
Clinical Profiles

ObjectiveTo elucidate current epidemiological, clinical profiles, and treatment of polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome.MethodsWe conducted a nationwide survey in 2015 using an established epidemiologic method. Data processing sheets were sent to all neurology and hematology specialist departments throughout Japan to identify patients with POEMS who were seen between April 2012 and March 2015.ResultsThe estimated number of patients with POEMS was 392 (95% confidence interval [CI] 320–464), and the prevalence was 0.3 per 100,000. Detailed clinical profiles were available for 167 patients. Median age at onset was 54 years (range, 21–84 years), and the ratio of male to female was 1.5. All patients showed polyneuropathy; 89% had monoclonal plasma cell proliferation; and 84% had elevated vascular endothelial growth factor level in whom pretreatment serum or plasma was available (n = 87). Other common features were skin changes (84%), edema/effusion (81%), and organomegaly (76%). A total of 160 patients were treated with any of the following: radiation, corticosteroids, melphalan, thalidomide, lenalidomide, bortezomib, or autologous stem cell transplantation. Primary therapeutic options were thalidomide (n = 86) and autologous stem cell transplantation (n = 71). Thirty-nine patients (24%) were initially treated with corticosteroid alone. The 10-year overall survival was 93% (95% CI 86%–96%).DiscussionThis study showed current epidemiologic and clinical status of POEMS syndrome in Japan. A quarter of patients were still inadequately treated with corticosteroid alone, whereas either autologous stem cell transplantation or immunomodulatory drugs improved the prognosis.

scholarly journals Long-term evaluation of physical improvement and survival of autologous stem cell transplantation in POEMS syndrome

Blood ◽  
2018 ◽  
Vol 131 (19) ◽  
pp. 2173-2176 ◽  
Author(s):  
Chikako Ohwada ◽  
Emiko Sakaida ◽  
Chika Kawajiri-Manako ◽  
Yuhei Nagao ◽  
Nagisa Oshima-Hasegawa ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Poems Syndrome ◽  
Term Evaluation

Peritransplant Complications and Long-Term Outcomes of Patients with Newly Diagnosed POEMS Syndrome after Upfront Autologous Stem Cell Transplantation

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2259-2259
Author(s):  
Jian Li ◽  
Ming-hui Duan ◽  
Wei Zhang ◽  
Chen Wang ◽  
Xu-fei Huang ◽  
...  
Keyword(s):  
Risk Factors ◽  
Stem Cell ◽  
Pleural Effusion ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Induction Therapy ◽  
Conditioning Regimen ◽  
Long Term Outcomes

Abstract Background : POEMS (Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal gammopathy and skin changes) syndrome is a rare plasma cell dyscrasia, without standardized therapeutic algorithm. Autologous stem cell transplantation (ASCT) has been reported to be effective, but can be complicated by excessive peritransplant events. Risk factors predictive for these complications are largely unknown. Moreover, long-term outcomes of ASCT have also not been studied comprehensively, especially in China. Methods : One-hundred and thirty eight patients, who met the diagnostic criteria proposed by Dispenzieri and underwent upfront ASCT at Peking Union Medical College Hospital between January 2005 and January 2016, were included in the current study. In these patients, 32 with severe end organ dysfunctions at diagnosis, received induction therapy (27 lenalidomide, 3 bortezomib and 2 melphalan based regimen; median 2, range 1-6 cycles) before ASCT. Clinical and laboratory information were reviewed, and outcomes were updated on July 1, 2016. Results : Median age at transplantation was 45 years (range 24-66 years), and 92 (67%) were male. Serum levels of vascular endothelial growth factor (VEGF) were dramatically elevated (median 4794, range 142-13867 pg/mL). Patients received pretransplant induction showed more pleural effusion, ascites, DLCO < 40% predicted and renal impairment (eGFR < 60 mL/min/1.73 m2). After induction, median value of serum VEGF was reduced from 4933 pg/mL to 804 pg/mL. Peripheral blood stem cells were mobilized by either cyclophosphamide with G-CSF (92%) or G-CSF alone (8%). Median dose of CD34+ cell collected was 2.58¡Á106/kg (0.77-10.90x106/kg). The conditioning regimen was melphalan 200 mg/m2 in 126 (91%), and the remaining patients received reduced dose (140 mg/m2). All collected CD34+ cells were infused. Severe peritransplant complications were recognized in 32 patients (16 engraftment syndrome, 5 cardiac events, 2 cerebrovascular events, 6 acute kidney injury and 3 treatment-related death). Baseline risk factors, identified through multivariate logistic regression, included age > 50 years (odds ratio [OR] 2.94, p = 0.025), time from symptom onset to transplant > 5 years (OR 4.89, p = 0.033) and pleural effusion (OR 3.17, p = 0.024). In contrast, induction therapy was associated with reduced complications (OR 0.19, p = 0.021), and more significant protective value was observed, if induction therapy was replaced by post-induction VEGF complete remission (CR) (OR 0.06, p = 0.012). After ASCT, most patients achieved systemic improvement, and the best responses were as follow: 50% hematological CR, 72% VEGF CR, 90% neurological response, and 65-86% responses of other involved organs. With median follow-up of 37 months (range 6-132 months), 6 patients died, and another 18 had disease progression. The 5-year overall survival and progression-free survival were 94% and 76%, respectively. Hematological (p = 0.008) and VEGF CRs (p = 0.021) were associated with superior PFS. Conclusion : Upfront ASCT is an effective therapy that can generate universal and durable responses in most POEMS patients. Pretransplant induction may make those cases with severe end organ dysfunction at diagnosis eligible for ASCT, and further reduce peritransplant complications. Disclosures No relevant conflicts of interest to declare.

Sign in / Sign up

Export Citation Format

Share Document