AL amyloidosis and other monoclonal immunoglobulin deposition diseases competence center
The AL amyloidosis and other monoclonal immunoglobulin deposition diseases competence center at the Necker-Enfants malades hospital is directed by Dr Laurent Frenzel. The center was labeled in July 2006. Amyloidosis is a complex disease due to the wide variety of symptoms presented by affected patients, which makes its management difficult. The number of new cases of AL amyloidosis in France is around 500 per year. The prognosis is closely linked to the rapid initiation of an effective treatment requiring a precise diagnosis of the type of amyloidosis and the assessment of the affected organs.
This reference center is affiliated with the MaRIH rare diseases healthcare network and the EuroBloodNet European reference network (ERN).
Keywords : AL amyloidosis, Randall disease, POEMS syndrome.
Medical
team
Dr Laurent Frenzel
MD, PhD
In case of emergency
- Management of patients with AL amyloidosis and other immunoglobulin deposition disorders
- Diagnosis
- Complications (cardiac, renal ..)
- Therapeutics (chemotherapy, immunotherapy …)
- Monitoring
- Organization of care in the hospital and at home with city-hospital relay
- Clinical studies with innovative therapies
- AL amyloidosis
- Non-amyloid monoclonal immunoglobulin deposition disease
- POEMS syndrome
- Light chain deposition disease
- Heavy chain deposition disease
- Acquired monoclonal Ig light chain-associated Fanconi syndrome
- Primary membranoproliferative glomerulonephritis
- C3 glomerulonephritis
- Cryoglobulinemias
- Peripheral neuropathy associated with monoclonal gammopathy
The AL amyloidosis and other immunoglobulin-related diseases competence center is integrated in the adult hematology department of Necker. This department includes an 8-bed protected hospitalization unit, a 12-bed intensive care unit, a 5-bed weekday hospital and a day hospital with an average of 20 patients per day. The medical team includes 6 permanent doctors, 2 contract doctors, 4 clinical directors and 4 medical interns. Given the active file, each unit has expertise in the management of pathologies related to the reference center. On the Necker site, the presence of a cardiology unit with 2 expert physicians and a conventional hospitalization unit and a cardiological intensive care unit (USIC) on the site of the Georges-Pompidou European hospital. On-site presence of an adult nephrology service, also expert in the pathologies related to the reference center. On-site presence of an adult intensive care unit with the necessary equipment for patient management.
Our research structure on our candidate site, the AL amyloidosis resource and competence center, consists of clinical research within the adult hematology department of Necker, with complete sharing of clinical studies between benign and malignant hematology. The clinical research team is composed of a clinical research coordinator and 4 full-time clinical research associates (CRA). In addition to the consultation, the environment includes a pediatric/adult clinical investigation center (CIC), a pediatric/adult day hospital and pediatric/adult hospitalization department. The center has been accredited for access to early phase 1 clinical studies, innovative therapies including cell and gene therapies.
Implementation of a home nursing supervision for the ambulatory management of treatments and side effects of patients in the framework of the reference center. Presence of a full-time social worker to assist with administrative procedures.
- IsaMYP – IFM (ongoing)
- NEOD001 – Industrial (ongoing)
Cohorts
- 10 patients included
- A prospective phase 2 trial of daratumumab in patients with previously treated systemic light-chain amyloidosis.
Roussel M, Merlini G, Chevret S, Arnulf B, Stoppa AM, Perrot A, Palladini G, Karlin L, Royer B, Huart A, Macro M, Morel P, Frenzel L, Touzeau C, Boyle E, Dorvaux V, Le Bras F, Lavergne D, Bridoux F, Jaccard A.
Blood. 2020 Apr 30;135(18):1531-1540. doi: 10.1182/blood.2019004369. PMID: 32108228. - Intraperitoneal bleeding as the initial presentation of Randall-type monoclonal immunoglobulin deposition disease with hepatic involvement.
Gkalea V, Amiot X, Jo Molina T, Frenzel L, Bachmeyer C.
Leuk Lymphoma. 2021 May;62(5):1263-1266. doi: 10.1080/10428194.2020.1856836. Epub 2020 Dec 10. PMID: 33300421. - POEMS syndrome : still an enigma ?
Hermine O, Frenzel L, Jaccard A.
Oncology (Williston Park). 2013 Dec;27(12):1260, 1262. PMID: 24624544.
Participation in congresses of the French Society of Hematology (SFH), American Society of Hematology (ASH) and national AL amyloidosis multidisciplinary consultation meetings (RCP)
Contact information
Necker-Enfants malades university hospital
> Adult hematology department
Hemophilia and hemorrhagic diseases treatment center
Adults and children
149 rue de Sèvres
75743 PARIS Cedex 15
> Welcome booklet – Adult patients
In Necker, the AL amyloidosis and other monoclonal immunoglobulin deposition diseases competence center in brief …
* data valid for 2021