The effect of diphenylhydantoin on the electroretinogram

2020

114

Disease Overview Immunoglobulin light chain amyloidosis is a clonal, nonproliferative plasma cell disorder in which fragments of immunoglobulin light or heavy chain are deposited in tissues. Clinical features depend on organs involved but can include heart failure with preserved ejection fraction, nephrotic syndrome, hepatic dysfunction, peripheral/autonomic neuropathy, and “atypical smoldering multiple myeloma or monoclonal gammopathy undetermined significance (MGUS).” Diagnosis Tissue biopsy stained with Congo red demonstrating amyloid deposits with apple‐green birefringence is required for diagnosis. Invasive organ biopsy is not required in 85% of patients. Verification that amyloid is composed of immunoglobulin light chains is mandatory. The gold standard is laser capture mass spectroscopy. Prognosis N‐terminal pro‐brain natriuretic peptide (NT‐proBNP), serum troponin T, and difference between involved and uninvolved immunoglobulin free light chain (FLC) values are used to classify patients into four groups of similar size; median survivals are 94.1, 40.3, 14.0, and 5.8 months. Therapy All patients with a systemic amyloid syndrome require therapy to prevent deposition of amyloid in other organs and prevent progressive organ failure. Stem cell transplant (SCT) is preferred, but only 20% of patients are eligible. Requirements for safe SCT include systolic blood pressure >90 mmHg, troponin T < 0.06 ng/mL and serum creatinine ≤1.7 mg/dL. Nontransplant candidates can be offered cyclophosphamide‐bortezomib‐dexamethasone or daratumumab‐containing regimens as it appears to be highly active in AL amyloidosis. Future Challenges Delayed diagnosis remains a major obstacle to initiating effective therapy prior to the development of end‐stage organ failure.

“…Registry data show reduced therapy‐related mortality and 77% five‐year survival. Outcomes were better at centers performing four AL transplants annually 88 …”

Section: Therapymentioning

confidence: 93%

“…Outcomes were better at centers performing four AL transplants annually. 88 The 10-year survival after SCT is 25%, and the 10-year survival rate was 53% for patients with a complete response (CR) to treatment. 89 Ten-year survival of all patients at our center is 43%.…”

Section: Therapymentioning

confidence: 99%

Immunoglobulin light chain amyloidosis: 2020 update on diagnosis, prognosis, and treatment

2020

114

“…Registry data show reduced therapy related mortality and 77% 5‐year survival. Outcomes were better at centers performing four AL transplants annually .…”

Section: Therapymentioning

confidence: 97%

Immunoglobulin light chain amyloidosis: 2016 update on diagnosis, prognosis, and treatment

2016

107

All patients with a systemic amyloid syndrome require therapy to prevent deposition of amyloid in other organs and prevent progressive organ failure of involved sites. Stem cell transplant (SCT) is preferred, but only 20% of patients are eligible. Requirements for safe SCT include systolic blood pressure >90 mmHg, troponin T <0.06 ng mL21, age <70 years, and serum creatinine 1.7 mg dL21. Nontransplant candidates can be offered melphalan-dexamethasone or cyclophosphamide-bortezomib-dexamethasone. Other combinations of chemotherapy with agents such as cyclophosphamide-thalidomide (or lenalidomide)-dexamethasone, bortezomib-dexamethasone, and melphalan-prednisone-lenalidomide have documented activity. Antibodies designed to dissolve existing amyloid deposits are under study for previously treated and untreated patients. Late diagnosis remains a major obstacle to initiating effective therapy. Am. J. Hematol., 2016. © 2016 Wiley Periodicals, Inc. Am. J. Hematol. 91:948-956, 2016. © 2016 Wiley Periodicals, Inc.

Immunoglobulin light chain amyloidosis: 2018 Update on diagnosis, prognosis, and treatment

2018

109

Upon completion of this educational activity, participants will be better able to: Master recognition of clinical presentations that should raise suspicion of amyloidosis. Understand simple techniques for confirming the diagnosis and providing material to classify the protein subunit. Recognize that a tissue diagnosis of amyloidosis does not indicate whether the amyloid is systemic or of immunoglobulin light chain origin. Understand the roles of the newly introduced chemotherapeutic and investigational antibody regimens for the therapy of light chain amyloidosis.