Rare Dermatology News
AL Amyloidosis is caused by the accumulation of an immunoglobulin light chain protein
Prevalence
25/100,000
Age of Onset
ICD-10
E85.9
Inheritance
Autosomal dominant
Autosomal recessive
Mitochondrial/Multigenic
X-linked dominant
X-linked recessive
5 Facts you should know
FACT
In AL amyloidosis, light chains come together to form amyloid deposits, which can cause serious damage to different organs
FACT
The kidneys are the most commonly affected organ in AL amyloidosis
FACT
Heart complications, which affect more than a third of AL patients, include heart failure and irregular heart beat
FACT
The most effective treatment is autologous bone marrow transplants with stem cell rescues
FACT
Two thirds of patients with AL amyloidosis are male and less than 5% of patients are under 40 years of age
Interest over time
Google searches
Common signs & symptoms
Fatigue
A subjective feeling of tiredness characterized by a lack of energy and motivation.
Weight loss
Reduction of total body weight.
Nephrotic syndrome
Nephrotic syndrome is a collection of findings resulting from glomerular dysfunction with an increase in glomerular capillary wall permeability associated with pronounced proteinuria. Nephrotic syndrome refers to the constellation of clinical findings that result from severe renal loss of protein, with Proteinuria and hypoalbuminemia, edema, and hyperlipidemia.
Hypertrophic cardiomyopathy
Hypertrophic cardiomyopathy (HCM) is defined by the presence of increased ventricular wall thickness or mass in the absence of loading conditions (hypertension, valve disease) sufficient to cause the observed abnormality.
Abnormality of the kidney
Abnormal pulmonary interstitial morphology - Abnormality of the lung parenchyma extending to the pulmonary interstitium and leading to diffuse pulmonary fibrosis.
Abnormal heart morphology
Any structural anomaly of the heart.
Current treatments
Medications
Chemotherapy
Some cancer drugs are used in AL amyloidosis to stop the growth of abnormal cells that produce the protein that forms amyloid. -Heart medications. If your heart is affected, you may need to take blood thinners to reduce the risk of clots. You may also need medications to control your heart rate. Drugs that increase urination can reduce the strain on your heart and kidneys.
Targeted therapies
For certain types of amyloidosis, drugs such as patisiran (Onpattro) and inotersen (Tegsedi) can interfere with the commands sent by faulty genes that create amyloid. Other drugs, such as tafamidis (Vyndamax, Vyndaqel) and diflunisal, can stabilize bits of protein in the bloodstream and prevent them from getting transformed into amyloid deposits.
Surgical and other procedures
Autologous blood stem cell transplant. This procedure involves collecting your own stem cells from your blood through a vein and storing them for a short time while you have high-dose chemotherapy. The stem cells are then returned to your body via a vein. This treatment is most appropriate for people whose disease isn't advanced and whose heart isn't greatly affected. -Dialysis. If your kidneys have been damaged by amyloidosis, you may need to start dialysis. This procedure uses a machine to filter wastes, salts and fluid from your blood on a regular schedule.
Organ transplant
If amyloid deposits have severely damaged your heart or kidneys, you might need surgery to replace those organs. Some types of amyloid are formed in the liver, so a liver transplant could stop that production.
Top Clinical Trials
| Title | Description | Phases | Status | Interventions | More Information |
|---|---|---|---|---|---|
| Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients | This is a single arm, open label study designed to evaluate the safety and efficacy of propylene glycol-free melphalan hydrochloride in patients with AL amyloidosis. Treatment will be comprised of propylene glycol-free melphalan hydrochloride administered intravenously at a dose of 70-100 mg/m2/day on Days -3 and -2 as conditioning prior to autologous stem cell transplantation. | Phase 2 | Recruiting | Drug: Propylene Glycol-Free Melphalan Hydrochloride | More Info |
| Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis | This is a single arm, Open-Label, Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis. | Phase 1/2 | Recruiting | Drug: ZN-d5 | More Info |
| COVID-19 VAX Booster Dosing in Patients With Hematologic Malignancies | To determine whether protective antibody levels increase after booster dosing with the Moderna COVID-19 vaccine in patients diagnosed with Hematologic Malignancies who have low antibody levels after a prior first vaccination with any of the SARS-CoV2 vaccines that were authorized for use in the USA. | Phase 2 | Recruiting | Drug: A single "booster" dose of the Moderna mRNA COVID-19 vaccine | More Info |
| Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Relapsed/Refractory Light Chain Amyloidosis Patients Previously Exposed to Daratumumab | This study will test the hypothesis that in patients with previous daratumumab exposure, combination therapy of daratumumab, pomalidomide, and dexamethasone (DPd) will yield higher complete remission (CR) rates in relapsed/refractory amyloidosis than historical pomalidomide/dexamethasone treatment. | Phase 2 | Recruiting | Drug: Daratumumab|Drug: Pomalidomide|Drug: Dexamethasone | More Info |
| Ixazomib In Combination With Cyclophosphamide And Dexamethasone for Newly Diagnosed AL Amyloidosis | The primary purpose of this study is to determine the safest dose of the medications and how well the combination is, or what is the "maximum tolerated dose" (MTD). | Phase 1|Phase 2 | Recruiting | Drug: Ixazomib|Drug: Cyclophosphamide|Drug: Dexamethasone | More Info |
| A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis | The primary purpose of this study is to determine if CAEL-101 improves the overall survival in Patients with cardiac AL Amyloidosis. | Phase 3 | Recruiting | Drug: CAEL-101|Other: Placebo|Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen | More Info |
| A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis | The primary purpose of this study is to determine if CAEL-101 improves the overall survival in Patients with cardiac AL Amyloidosis. | Phase 3 | Recruiting | Drug: CAEL-101|Other: Placebo|Drug: cyclophosphamide, bortezomib, and Dexamethasone (CyBorD) regimen | More Info |
| Ixazomib Maintenance Study in Patients With AL Amyloidosis | The purpose of this study is to learn if Ixazomib maintenance treatment (chemotherapy) works to control the disease. | Phase 2 | Recruiting | Drug: Ixazomib|Drug: Dexamethasone | More Info |
| APG-2575 in Combination With Novel Therapeutic Regimens in Subjects With Relapsed or Refractory Multiple Myeloma | This is a Phase Ib/II, open-label, multi-center study evaluating the safety, tolerability, efficacy, and PK/ Pharmacodynamics of APG-2575 in combination with Pd/DRd in patients with relapsed/refractory (RR) multiple myeloma (MM) | Phase 1/2 | Recruiting | Drug: APG-2575+ Pd|Drug: APG-2575 + DRd | More Info |
| A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis | A Phase 3 study to evaluate the efficacy and safety of birtamimab plus standard of care compared to placebo plus standard of care in patients with AL amyloidosis in Mayo Stage IV. | Phase 3 | Recruiting | Drug: Birtamimab|Other: Placebo|Drug: Standard of Care Chemotherapy | More Info |
Top Treatments in Research
| Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
|---|---|---|---|---|---|
| propylene glycol-free melphalan hydrochloride | Propylene Glycol-Free melphalan HCL for Injection (PGF-Mel) is a new formulation that incorporates Captisol, a specially modified cyclodextrin, to improve melphalan stability. | Phase 2 | Spectrum Pharamceuticals | More Info | More Info |
| ZN-d5 | A potent, selective, and orally bioavailable BCL-2 inhibitor | Phase 1/2 | K-Group Alpha, Inc. | More Info | More Info |
| Moderna COVID-19 vaccine | mRNA vaccine | Phase 2 | Moderna | More Info | More Info |
| Daratumumab/Pomalidomide/Dexamethasone | Daratumumab is a human mab that binds to a unique cd38 epitope on cd38-expressing cells. Pomalidomide is an immunomodulatory and antineoplastic agent. | Phase 2 | Weill Medical College of Cornell University | More Info | More Info |
| Ixazomib/Cyclophosphamide/Dexamethasone | Ixazomib is a second-generation proteasome inhibitor | Phase 1/2 | Icahn School of Medicine at Mount Sinai Collaborator: Millennium Pharmaceuticals, Inc. | More Info | More Info |
| CAEL-101 | CAEL-101 is a monoclonal antibody that acts by binding to amyloid light chain fibrils and promoting their clearance from affected tissues | Phase 3 | Caelum Biosciences, Inc. | More Info | More Info |
| CAEL-101 | CAEL-101 is a monoclonal antibody that acts by binding to amyloid light chain fibrils and promoting their clearance from affected tissues | Phase 3 | Caelum Biosciences, Inc. | More Info | More Info |
| Ixazomib | Ixazomib is a second-generation proteasome inhibitor | Phase 2 | Memorial Sloan Kettering Cancer Center | More Info | More Info |
| APG-2575+ Pd | A novel, orally administered Bcl-2‒selective inhibitor | Phase 1/2 | Ascentage Pharma Group | More Info | More Info |
| Birtamimab | Birtamimab (NEOD001), is an investigational monoclonal antibody designed to specifically and selectively target and clear the amyloid that accumulates and causes organ dysfunction and failure in patients with AL amyloidosis | Phase 3 | Prothena Biosciences | More Info | More Info |
