Improved Outcomes After Autologous Hematopoietic Cell Transplantation for Light Chain Amyloidosis: A Center for International Blood and Marrow Transplant Research Study

D’Souza, Anita; Dispenzieri, Angela; Wirk, Baldeep; Zhang, Mei Jie; Huang, Jiaxing; Gertz, Morie A.; Kyle, Robert A.; Kumar, Shaji; Comenzo, Raymond L.; Gale, Robert Peter; Lazarus, Hillard M.; Savani, Bipin N.; Cornell, Robert F.; Weiss, Brendan M.; Vogl, Dan T.; Freytes, César O.; Scott, Emma C.; Landau, Heather; Moreb, Jan S.; Costa, Luciano J.; Ramanathan, Muthalagu; Callander, Natalie S.; Kamble, Rammurti T.; Olsson, Richard F.; Ganguly, Siddhartha; Nishihori, Taiga; Kindwall‐Keller, Tamila L.; Wood, William A.; Mark, Tomer M.; Hari, Parameswaran

doi:10.1200/jco.2015.62.4015

Cited by 171 publications

(143 citation statements)

References 31 publications

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“…Refinement of eligibility criteria over time resulted in a progressive reduction of TRM to as low as 5%. 57 However, this improvement was limited to patients without cardiac involvement. 57 Cardiac biomarkers play a central role in the assessment of • Low-dose combination regimens…”

Section: Chemotherapymentioning

confidence: 99%

“…60 Center experience also affects TRM, which is significantly higher (7% vs 3%) at institutions where fewer than 4 transplants are performed every year. 57 The hematologic response rate to ASCT exceeds 70%, with 35% to 37% of patients obtaining CR. 57,61 The Boston University investigators recently updated their experience with ASCT.…”

Section: What's New In Amyloidosis 163mentioning

confidence: 99%

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What is new in diagnosis and management of light chain amyloidosis?

Palladini

Merlini

2016

Blood

196

169

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Light chain (AL) amyloidosis is caused by a usually small plasma cell clone producing a misfolded light chain that deposits in tissues. Survival is mostly determined by the severity of heart involvement. Recent studies are clarifying the mechanisms of cardiac damage, pointing to a toxic effect of amyloidogenic light chains and offering new potential therapeutic targets. The diagnosis requires adequate technology, available at referral centers, for amyloid typing. Late diagnosis results in approximately 30% of patients presenting with advanced, irreversible organ involvement and dying in a few months despite modern treatments. The availability of accurate biomarkers of clonal and organ disease is reshaping the approach to patients with AL amyloidosis. Screening of early organ damage based on biomarkers can help identify patients with monoclonal gammopathy of undetermined significance who are developing AL amyloidosis before they become symptomatic. Staging systems and response assessment based on biomarkers facilitate the design and conduction of clinical trials, guide the therapeutic strategy, and allow the timely identification of refractory patients to be switched to rescue therapy. Treatment should be risk-adapted. Recent studies are linking specific characteristics of the plasma cell clone to response to different types of treatment, moving toward patient-tailored therapy. In addition, novel anti-amyloid treatments are being developed that might be combined with anti-plasma cell chemotherapy.

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Section: Chemotherapymentioning

confidence: 99%

Section: What's New In Amyloidosis 163mentioning

confidence: 99%

What is new in diagnosis and management of light chain amyloidosis?

Palladini

Merlini

2016

Blood

196

169

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show abstract

“…Autologous stem cell transplantation (ASCT) is considered the treatment of choice, when applicable, given its superior long-term survival. 1,2 However, for non-ASCT eligible patients, therapy selection based on baseline disease characteristics has not been well-developed. The lack of guidelines for treatment selection at diagnosis may lead to the selection of inappropriate treatment that would lead to irreversible organ damage and a shorter survival.…”

Section: Introductionmentioning

confidence: 99%

Immunoparesis status in immunoglobulin light chain amyloidosis at diagnosis affects response and survival by regimen type

et al. 2016

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“…The second-line treatment is based on thalidomide [42]. Certain patients can undergo a consolidation with high-dose melphalan chemotherapy followed by autologous stem cell transplantation [43,44]. Stage iii patients present a therapeutic challenge because of an extremely poor prognosis.…”

Section: Treatment Of Amyloidosismentioning

confidence: 99%

Monoclonal gammopathy with renal significance

Sysakiewicz¹,

Czyż²,

Ksieniewicz³

et al. 2016

Medical Research Journal

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Monoclonal gammopathy of unknown significance (MGUS) is a benign condition that carries a risk of progression to haematological malignancy. It is accepted that MGUS should not be treated until progression to multiple myeloma or another lymphoid malignancy. Recently, growing evidence has started to show that even small monoclonal clones can be responsible for renal impairment. Long-term observation of patients with monoclonal gammopathy and abnormal renal function showed that this condition can significantly affect renal and overall survival. Patients with monoclonal gammopathy with renal impairment have also higher risk of relapse after kidney transplantation. Among patients with monoclonal gammopathy of unknown significance there is a group of defined monoclonal component-related diseases, which includes:light-chain amyloidosis, monoclonal immunoglobulin deposition disease, crystal-storing histiocytosis, cryoglobulinaemias, and some others. They can be diagnosed on the base of clinical features and on histological examination. In patients with monoclonal protein and deposition of fragments or whole particle of monoclonal immunoglobulin with distinct localisation and substructural organisation can be found.The treatment strategy is targeting of B cell clones, which requires administration of chemotherapeutics or other medications that are used for the treatment of lymphoid malignancy or myeloma. The choice of therapeutic agent should take into account the current kidney status. Treatment of renal disease should not differ from other patients with similar conditions not related to monoclonal protein. The expert opinion is that the presence of monoclonal gammapathy is not a contraindication to kidney transplantation.

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Improved Outcomes After Autologous Hematopoietic Cell Transplantation for Light Chain Amyloidosis: A Center for International Blood and Marrow Transplant Research Study

Abstract: Post-transplantation survival in AL has improved, with a dramatic reduction in early post-transplantation mortality and excellent 5-year survival. The risk-benefit ratio for autotransplantation has changed, and randomized comparison with nontransplantation approaches is again warranted.

Cited by 171 publications

References 31 publications

What is new in diagnosis and management of light chain amyloidosis?

What is new in diagnosis and management of light chain amyloidosis?

Immunoparesis status in immunoglobulin light chain amyloidosis at diagnosis affects response and survival by regimen type

Monoclonal gammopathy with renal significance

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