The prognosis in breast cancer is generally poor for women with unresectable, locally advanced disease (inflammatory cancer or more than 10 positive lymph nodes) and for those with metastases. Autologous bone marrow transplantation - removing bone marrow from multiple sites under general anesthesia, giving high-dose chemotherapy with or without total body radiation, and reinfusing the bone marrow intravenously - is being tried in some of these patients.

Recombinant human deoxyribonuclease I (rhDNase; dornase alfa; Pulmozyme - Genentech), an enzyme that hydrolyzes extracellular DNA, is now available as a purified solution to decrease the viscosity of sputum in patients with cystic fibrosis. It requires a nebulizer to produce an aerosol mist for inhalation.

High-dose chemotherapy, with or without total body radiation, followed by bone marrow transplantation to restore bone marrow function has been used widely in the treatment of malignant diseases in recent years (Medical Letter, 34:79, 1992; 37:25, 1995). Now, however, instead of using bone marrow itself to restore function destroyed by chemotherapy and radiation, many centers infuse hematopoietic stem cells harvested from peripheral blood. Most stem cell transplants have been autologous; allogeneic stem cells have been tried in a small number of patients (WI Bensinger et al, Blood,...

Anastrozole (Arimidex - Zeneca), a selective nonsteroidal aromatase inhibitor, has been approved by the US Food and Drug Administration (FDA) for treatment of postmenopausal women with advanced breast cancer that has progressed during treatment with tamoxifen (Nolvadex, and others).

Tamoxifen, anti-estrogen used for many years in the treatment of breast cancer, has now been approved by the FDA for reducing the incidence of breast cancer in women at increased risk of developing the disease.

A "Dear Colleague" letter from the UK's Committee on Safety of Medicines advised physicians, on the basis of unpublished data, not to use paroxetine (Paxil in the US; Seroxat in the UK) to treat children and adolescents with depression because of possible increased risk of suicidal behavior. This review describes the efficacy and safety of SSRIs in children.

The FDA has approved ruxolitinib (Jakafi – Incyte), a janus-associated kinase (JAK) inhibitor, for treatment of myelofibrosis. Jakafi is the first JAK inhibitor to be approved for any indication and the only drug approved for treatment of myelofibrosis.

The FDA has approved sebelipase alfa (Kanuma – Alexion), a recombinant form of lysosomal acid lipase (LAL), for enzyme replacement therapy in patients with LAL deficiency. Sebelipase alfa is the first drug to be approved in the US for treatment of LAL deficiency, a rare autosomal recessive storage disease.

Olaratumab (Lartruvo – Lilly), a platelet-derived growth factor receptor alpha (PDGFR-α) blocking monoclonal antibody, has received accelerated approval from the FDA for use in combination with the anthracycline doxorubicin for first-line treatment of adults with soft-tissue sarcoma histologic subtypes considered susceptible to anthracyclines. Approval is limited to locally advanced or metastatic soft-tissue sarcomas that are not amenable to curative radiotherapy or surgery, and is contingent on verification of clinical benefit in a confirmatory phase 3 trial.

The FDA has approved setmelanotide (Imcivree – Rhythm), a subcutaneously injected melanocortin 4 (MC4) receptor agonist, for chronic weight management in patients ≥6 years old with obesity due to pro-opiomelanocortin (POMC) deficiency, proprotein subtilisin/kexin type 1 (PCSK1) deficiency, or leptin receptor (LEPR) deficiency.