High‐dose therapy and autologous transplantation for POEMS Syndrome: effective, but how to optimise?

Tomkins, Oliver; Keddie, Stephen; Lunn, Michael P.; D’Sa, Shirley

doi:10.1111/bjh.16057

Cited by 11 publications

(17 citation statements)

References 10 publications

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“…The Mayo group has described high levels of engraftment syndrome (ES) of up to 40% 18 but this may be due the use of granulocyte-macrophage colony-stimulating factor (GM-CSF) instead of granulocyte colony-stimulating factor (G-CSF) for post-ASCT engraftment, 19 as other groups have reported lower rates of 6%-23%. 3,13,20,21 It has been postulated that cyclophosphamide may reduce of ES, due to immune-modulation by this drug. 13 Increased peri-engraftment complications including cardiac events, renal impairment infection are associated with age over 50 years, time from symptoms to ASCT more than five years, presence of pleural effusions, and lack of achievement of a CR V post induction.…”

Section: Autologous Stem Cell Transplantationmentioning

confidence: 99%

“…The Mayo group has described high levels of engraftment syndrome (ES) of up to 40% 18 but this may be due the use of granulocyte‐macrophage colony‐stimulating factor (GM‐CSF) instead of granulocyte colony‐stimulating factor (G‐CSF) for post‐ASCT engraftment, 19 as other groups have reported lower rates of 6%–23% 3,13,20,21 . It has been postulated that cyclophosphamide may reduce incidence of ES, due to immune‐modulation by this drug 13 .…”

Section: Front‐line Therapymentioning

confidence: 99%

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Evidence‐based medical treatment of POEMS syndrome

et al. 2022

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I N TRODUC TIONPOEMS syndrome is a rare multisystem paraneoplastic disorder characterised by Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal protein and Skin changes. The exact incidence is unknown. Patients may have diverse clinical presentations which are not found in the acronym, including extra-vascular overload, thrombosis and elevated raised serum vascular endothelial growth factor (VEGF). Full diagnostic criteria are outlined in Table 1. The diagnosis of POEMS syndrome is confirmed when both of the mandatory major criteria, one of the three other major criteria, and one of the six minor criteria are present. The underlying pathogenesis is not fully understood; however, an underlying slowly progressive low-level plasma cell dyscrasia is central alongside elevated VEGF. Most patients do not meet criteria for myeloma, with less than 5% plasma cell infiltration being the median infiltrate in a series of 67 patients, 1 on a background of polytypic plasma cells, as well as megakaryocyte hyperplasia, clustering and atypia. Delayed diagnosis is very common, of up to two years, 2 which may lead to organ dysfunction and subsequent adverse outcomes. 3 However, treatment options are highly effective at eradicating the underlying clone. Due to the rarity of POEMS, there are limited trial data to guide treatment decisions and no international consensus guidelines. Data are largely gleaned from retrospective case series. Therapeutic options are adapted from those used in multiple myeloma and solitary plasmacytoma. Other reviews have looked at the overall management of POEMS 4 ; here we focus specifically on medical treatment options, and present the first systematic review of current available data. Treatment aimsTreatment is primarily directed at eradicating the underlying plasma cell dyscrasia. For most patients, treatment is highly effective in halting disease progression. The aims of treatment are prolongation of overall survival (OS), increased duration of remission (progression-free survival, PFS) and

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Section: Autologous Stem Cell Transplantationmentioning

confidence: 99%

Section: Front‐line Therapymentioning

confidence: 99%

Evidence‐based medical treatment of POEMS syndrome

et al. 2022

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“…In a recent study from MD Anderson Cancer Center, this approach was associated with 0% transplant‐related mortality, 100% clinical response rate, and 4‐year and 10‐year OS of 100 and 80%, respectively, highlighting the safety and efficacy of this approach 38 . Other investigators have reported similar results with auto‐HCT from several institutions 39‐41 …”

Section: Therapymentioning

confidence: 82%

POEMS syndrome: A multisystem clonal disorder

Ali

Qazilbash

2020

European J of Haematology

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Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes (POEMS) syndrome is a rare condition defined by monoclonal plasma cell disorder, peripheral neuropathy, and other systemic symptoms. The pathophysiology of POEMS syndrome is unknown, but the overproduction of vascular endothelial growth factor (VEGF) appears to be an important contributory element. The diagnosis of POEMS syndrome requires the presence of both mandatory criteria (ie, polyneuropathy and a monoclonal plasma cell disorder), at least one major criterion (ie, osteosclerotic bone lesions, Castleman disease, or elevated serum or plasma levels of vascular endothelial growth factor), and at least one of the six minor criteria. POEMS syndrome lacks a standard treatment, but patients with limited sclerotic bone lesions are typically treated with radiation therapy. In contrast, those with widespread lesions receive chemotherapy and hematopoietic stem cell transplantation.

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“…Overall, ASCT is a safe and effective choice in many patients and can provide durable response in about 90% of eligible patients. Different studies and retrospective analysis confirmed high rates of overall survival (OS) and progression free survival (PFS), 98% at one year, following ASCT [ 51 , 52 , 69 , 70 ], with a significantly lower rate of relapse [ 69 , 70 ] in a median of 5 months post treatment [ 68 , 71 ]. Relapses of patients after ASCT is positively correlated with immunoglobulin G-λ monoclonal gammopathy presence of FDG-avid lesions on baseline PET scan and inability to achieve hematologic response with ASCT [ 72 ].…”

Section: Targeting the Clonementioning

confidence: 99%

Recent Advances in the Treatment and Supportive Care of POEMS Syndrome

Zerdan

George

Bunting

et al. 2022

JCM

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POEMS is a rare clonal plasma cell disorder characterized by multi-systemic features that include demyelinating peripheral neuropathy, organomegaly, endocrinopathy, presence of monoclonal proteins (M-protein), and skin changes. Even though the pathophysiology is poorly understood, recent studies suggest that both clonal and polyclonal plasmacytosis leading to the production of pro-inflammatory cytokines and angiogenic mediators play the central role. These mediators including vascular endothelial growth factor (VEGF) are the driving forces of the syndrome. The diagnosis of POEMS is not always straight forward and often the diagnosis is delayed. It is based on fulfilling mandatory criteria of polyradiculoneuropathy and monoclonal protein and the presence of one major criterion (Castleman disease, sclerotic bone lesions, or elevated VEGF), and at least one minor criterion. Due to the presence of neuropathy, it can be confused with chronic inflammatory demyelinating polyradiculopathy (CIDP), and if thrombocytosis and splenomegaly are present, it can be confused with myeloproliferative neoplasms. Due to the rarity of the syndrome, clear guidelines for treatment are still lacking. Immediate treatment targeting the underlying plasma cell proliferation results in a dramatic response in most patients. The key is early diagnosis and immediate anti-plasma cell directed therapy for the best clinical outcomes. For patients with disseminated disease as defined by bone marrow involvement or more than three osteosclerotic bone lesions, high-dose chemotherapy with autologous hematopoietic stem cell transplant (ASCT) yields durable responses and is the preferred treatment in eligible patients. For patients with localized bony disease, radiotherapy has proven to be very effective. Lenalidomide and dexamethasone is a proven therapy in patients ineligible for ASCT. In this review article, we tackle the diagnostic approach and discuss the latest treatment modalities of this rare debilitating disease.

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High‐dose therapy and autologous transplantation for POEMS Syndrome: effective, but how to optimise?

Cited by 11 publications

References 10 publications

Evidence‐based medical treatment of POEMS syndrome

Evidence‐based medical treatment of POEMS syndrome

POEMS syndrome: A multisystem clonal disorder

Recent Advances in the Treatment and Supportive Care of POEMS Syndrome

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