Treatment and prognosis

Precise diagnosis and prognosis assessment are crucial for the choice of treatment and expected goals hereof.

AL amyloidosis can be treated with chemotherapy, which has no effect on other types. Younger, suitable patients without severe co-morbidity or multi-organ involvement, including absence of significant cardiac involvement, can be offered high-dosage chemotherapy with support from the patient’s own blood stem cells. Due to frequently widespread and serious amyloid manifestations, the majority of AL amyloidosis patients will not be candidates for intensive chemotherapy. Bortezomib-based regimes have generally shown high efficiency on AL amyloidosis and have an effect that seems to be comparable to intensive chemotherapy. Other effective drugs, other than cytostatics, are immunomodulatory drugs (ImiDs), including particularly lenalidomid and new proteasome inhibitors.

New treatments, including monoclonal antibodies that target the clonal plasma cells, are expected to show high activity for AL amyloidosis and are therefore expected to also challenge the need for intensive chemotherapy.

Other antibodies and drugs are being developed and tested that directly target the amyloid substance, for both AL amyloidosis and ATTR amyloidosis. These treatments aim to promote resolution of the amyloid, resulting in improved organ function. There is great optimism surrounding the perspectives of these treatments.

For all patients with systemic amyloidosis, optimal supporting treatment is crucial, and it should be performed in close cooperation between relevant specialties. This is ensured at AmyC OUH through a direct interdisciplinary consultation, where the patient meets e.g. hematologist and cardiologist at the same consultation.

There are further a number of drugs that are completely or partially contraindicated for systemic amyloidosis. This emphasizes the importance of the patients being assessed and followed by specialist doctors with extensive insight into and interest in the illness.

Systemic amyloidosis with multi-organ involvement is associated with a serious prognosis. This is particularly true in cases with significant cardiac involvement. However, with the new treatment options, the prognosis is improving significantly.