Response of factor X deficiency to darutumumab in the treatment of AL amyloidosis: a novel finding

2021 ◽  
Vol 14 (4) ◽  
pp. e240631
Author(s):  
Ethan Mar ◽  
Kerry Taylor ◽  
Peter Mollee
Keyword(s):  
Quality Of Life ◽  
Case Report ◽  
Light Chain ◽  
Al Amyloidosis ◽  
Factor X ◽  
Haematological Response ◽  
Factor X Deficiency ◽  
First Case ◽  
Complete Haematological Response

We report a case of progressive light-chain amyloidosis (otherwise known as AL amyloidosis) with acquired factor X (aFX) deficiency with a complete haematological response and rapid normalisation of FX levels following daratumumab monotherapy. To our knowledge, this is the first case report documenting successful treatment with daratumumab of aFX deficiency secondary to AL amyloidosis. The patient responded well to this therapy, with excellent symptomatic and quality of life improvements as well as a reduction in bleeding manifestations. This case highlights the value in considering daratumumab treatment when AL amyloidosis is complicated by FX deficiency.

Acquired factor X deficiency in patients with amyloid light-chain amyloidosis: incidence, bleeding manifestations, and response to high-dose chemotherapy

Blood ◽  
2001 ◽  
Vol 97 (6) ◽  
pp. 1885-1887 ◽  
Author(s):  
Elie B. Choufani ◽  
Vaishali Sanchorawala ◽  
Timothy Ernst ◽  
Karen Quillen ◽  
Martha Skinner ◽  
...  
Keyword(s):  
Light Chain ◽  
Medical Center ◽  
Bleeding Complications ◽  
Plasma Cell Dyscrasia ◽  
High Dose ◽  
Al Amyloidosis ◽  
Related Factor ◽  
Factor X ◽  
Factor X Deficiency ◽  
Amyloid Light Chain

Acquired deficiency of factor X occurs in patients with systemic amyloid light-chain (AL) amyloidosis, presumably due to adsorption of factor X to amyloid fibrils. Of 368 consecutive patients with systemic AL amyloidosis evaluated at Boston Medical Center, 32 patients (8.7%) had factor X levels below 50% of normal. Eighteen of these patients (56%) had bleeding complications, which were more frequent and severe in the 12 patients below 25% of normal; 2 episodes were fatal. Ten factor X–deficient patients received high-dose melphalan chemotherapy followed by autologous stem cell transplantation. Of 7 patients alive 1 year after treatment, 4 had a complete hematologic response, and all 4 experienced improvement in their factor X levels. One of 2 additional patients with partial hematologic responses had improvement in factor X. Thus, aggressive treatment of the underlying plasma cell dyscrasia in AL amyloidosis can lead to the amelioration of amyloid-related factor X deficiency.

scholarly journals Acquired factor X deficiency in light-chain (AL) amyloidosis is rare and associated with advanced disease

2019 ◽  
Vol 12 (1) ◽  
pp. 10-14 ◽  
Author(s):  
Gina Patel ◽  
Parameswaran Hari ◽  
Aniko Szabo ◽  
Lisa Rein ◽  
Lisa Baumann Kreuziger ◽  
...  
Keyword(s):  
Light Chain ◽  
Advanced Disease ◽  
Al Amyloidosis ◽  
Factor X ◽  
Factor X Deficiency

scholarly journals FOCUSED REVIEW ON NUTRITIONAL STATUS OF PATIENTS WITH IMMUNOGLOBULIN LIGHT CHAIN AMYLOIDOSIS

2021 ◽  
Author(s):  
Shana Souza Grigoletti ◽  
Priccila Zuchinali ◽  
Emilie Lemieux-Blanchard ◽  
Stephanie Bechard ◽  
Bernard Lemieux ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Weight Loss ◽  
Nutritional Status ◽  
Light Chain ◽  
Complex Disease ◽  
Negative Impact ◽  
Al Amyloidosis ◽  
Immunoglobulin Light Chain ◽  
Negatively Associated

Background: Immunoglobulin light chain (AL) amyloidosis is a complex disease marked by a poor clinical portrait and prognosis generally leading to organ dysfunction and shortened survival. We aimed to review the available evidence on whether AL amyloidosis can lead to malnutrition, thus having a negative impact on quality of life (QoL) and survival. Materials: We searched Pubmed with no restrictions to the year of publication or language. Retrospective or prospective, observational, and interventional studies that reported data regarding AL amyloidosis and nutritional status were included. Results: From 62 articles retrieved, 23 were included. Malnutrition was prevalent in up to 65% of patients with AL Amyloidosis. Prevalence of weight loss of 10% or more ranged from 6 to 22% of patients, while a body mass index of < 22 kg/m2 was found in 22 to 42%. Weight loss, lower BMI and other indicators of poor nutritional status were negatively associated with quality of life and survival. Only one RCT focused on nutritional counselling was found and reported positive results on patients' QoL and survival. Conclusion Despite inconsistencies across assessment criteria, the available data reveal that weight loss and malnutrition are common features in patients with AL amyloidosis. This review reinforces the premise that an impaired nutritional status can be negatively associated with QoL and survival in patients with AL amyloidosis, and therefore should be further investigated.

Nutritional status independently affects quality of life of patients with systemic immunoglobulin light-chain (AL) amyloidosis

2011 ◽  
Vol 91 (3) ◽  
pp. 399-406 ◽  
Author(s):  
Riccardo Caccialanza ◽  
Giovanni Palladini ◽  
Catherine Klersy ◽  
Emanuele Cereda ◽  
Chiara Bonardi ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Nutritional Status ◽  
Light Chain ◽  
Al Amyloidosis ◽  
Immunoglobulin Light Chain

scholarly journals Case Report: Management of Primary Tracheobronchial Light Chain Amyloidosis in a Patient With Biclonal Gammopathy Using a Systemic Bortezomib-Based Regimen

2021 ◽  
Vol 8 ◽  
Author(s):  
Wei Yan ◽  
Peng Li ◽  
Cen Wu ◽  
Chuming Zhou ◽  
Aijun Liao ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Light Chain ◽  
Local Treatment ◽  
Wall Thickening ◽  
Current View ◽  
Al Amyloidosis ◽  
Airway Patency ◽  
Biclonal Gammopathy ◽  
Localized Form

Primary tracheobronchial light chain (AL) amyloidosis is a rare and heterogeneous disease characterized by the buildup of amyloid deposits in the airway mucosa. Although its treatment remains challenging, the current view is that the localized form can be treated conservatively due to its slow progression. While radiotherapy has proven effective in treating localized form of the disease, some patients do not respond to local treatment and continue to experience poor quality of life, highlighting the need to explore additional treatment strategies. In this report, we discuss a case of primary tracheobronchial AL amyloidosis with biclonal gammopathy (IgA κ and IgG κ) in a 46-year-old man who was transferred to our hospital due to dyspnea progression over the preceding 3 years. Chest computed tomography revealed irregular tracheobronchial stenosis with wall thickening, and histological examination of the bronchial biopsies confirmed the diagnosis of endobronchial AL amyloidosis. Owing to the poor effect of radiation therapy and treatments for improving airway patency, he was treated with a systemic chemotherapy regimen [cyclophosphamide-bortezomib-dexamethasone (CyBorD)]. We observed substantial improvements in his dyspnea, highlighting the potential of systemic therapy to improve quality of life of patients with tracheobronchial AL amyloidosis. However, the long-term pathological changes associated with local bronchial lesions require further investigation.

Nutritional counseling improves quality of life and preserves body weight in systemic immunoglobulin light-chain (AL) amyloidosis

Nutrition ◽  
2015 ◽  
Vol 31 (10) ◽  
pp. 1228-1234 ◽  
Author(s):  
Riccardo Caccialanza ◽  
Giovanni Palladini ◽  
Emanuele Cereda ◽  
Chiara Bonardi ◽  
Paolo Milani ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Body Weight ◽  
Light Chain ◽  
Al Amyloidosis ◽  
Immunoglobulin Light Chain ◽  
Nutritional Counseling

scholarly journals Cardiac biomarkers and health‐related quality of life in patients with light chain ( AL ) amyloidosis

2018 ◽  
Vol 185 (5) ◽  
pp. 998-1001
Author(s):  
Kristen L. McCausland ◽  
Tiffany P. Quock ◽  
Avery A. Rizio ◽  
Martha S. Bayliss ◽  
Michelle K. White ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Light Chain ◽  
Cardiac Biomarkers ◽  
Al Amyloidosis ◽  
Health Related Quality ◽  
Related Quality ◽  
Health Related
Sign in / Sign up

Export Citation Format

Share Document