- N Engl J Med 2022; 387:775-777
For patients with rare diseases, connecting virtually with specialists across state lines can be the only option for excellent care. So why are state-licensing restrictions being enforced more aggressively than ever?
- N Engl J Med 2022; 387:488-491
Many children born with sickle-cell disease in sub-Saharan Africa will die before their fifth birthday. Opportunities exist to expand the distribution, use, and oversight of effective medication.
- N Engl J Med 2022; 386:2471-2481
A total of 12 pediatric patients with diffuse intrinsic pontine glioma were treated with a direct infusion of an oncolytic virus, followed by radiotherapy. The tumor size was reduced in 9 patients, and disease was stable over a median follow-up of 18 months in 8 patients.
- N Engl J Med 2022; 386:2295-2302
Three patients with Schimke immuno-osseous dysplasia who underwent sequential bone marrow and kidney transplantation continued to have normal renal function without immunosuppression at 22 to 34 months.
- N Engl J Med 2022; 386:2332-2333
Stunning advances in human transplantation have been tempered by the immunologic consequences of histoincompatibility between donor and recipient — namely, allograft rejection after solid-organ transplantation and graft-versus-host disease (GVHD) after hematopoietic stem-cell (bone marrow) transplantation (HSCT). The separation of GVHD from the graft-versus-tumor effect and the induction of functional immune...
- N Engl J Med 2022; 386:2059-2060
Autopsy material from a child who died from an acute flaccid myelitis–like illness showed EV-D68 RNA and protein in anterior horn cells and their axons, strongly implicating the virus as the cause of the disorder.
- N Engl J Med 2022; 386:2011-2023
In part 1 of a phase 2–3 trial, a 50-μg dose of mRNA-1273 vaccine was safe and immunogenic. In part 2, nearly 4000 6-to-11-year-olds received two doses of vaccine or placebo and were followed for a median of 82 days. The vaccine had mainly mild adverse effects and was immunogenic...
- N Engl J Med 2022; 386:1873-1875
Medicine promotes a logically inconsistent message regarding SIDS: authoritative statements imply that SIDS is a consequence of unsafe sleep in biologically normal children, but there is vague acknowledgment that important biologic factors are involved in these deaths.
- N Engl J Med 2022; 386:1922-1931
Childhood brain tumors were previously classified by histologic appearance and location. A new classification includes genetic changes, which have prognostic significance and may lead to therapeutic targets. This review focuses on pediatric gliomas, ependymomas, and medulloblastomas.
- N Engl J Med 2022; 386:e50
A 14-year-old boy with acute lymphoblastic leukemia who had been receiving maintenance chemotherapy presented with acute blurry vision. Examination showed premacular hemorrhages in both retinas.
- N Engl J Med 2022; 386:1615-1626
Respiratory syncytial virus is a major cause of illness in infants. In this randomized, double-blind, placebo-controlled trial, the safety and immunogenicity of a bivalent RSV prefusion F protein–based vaccine was assessed in pregnant women and their infants. Anti-RSV antibodies were elicited with efficient transplacental transfer.
- N Engl J Med 2022; 386:1638-1645
Development and public acceptance of vaccines are crucial for disease control. This review covers the process of evaluating vaccines for safety and efficacy, authorization pathways, and factors that advisory bodies consider when establishing vaccine recommendations.
- N Engl J Med 2022; 386:1282-1283
Attempts to control malaria have been making good progress in South America, and two countries, Paraguay and Argentina, have recently been certified by the World Health Organization (WHO) as having achieved elimination of malaria. Brazil has the highest incidence of malaria in South America,1 with 143,381 indigenous cases...
- N Engl J Med 2022; 386:1244-1253
The hypnozoite form of Plasmodium vivax malaria can lead to recrudescent infection months after primary infection. Primaquine is used as prophylaxis to prevent such relapses. In this report, a higher total dose of primaquine (7.0 mg per kilogram over 14 days) was found to be more efficacious than...
- N Engl J Med 2022; 386:1184-1186
Gene therapy for disorders affecting muscle, such as Duchenne’s muscular dystrophy and myotubular myopathy, has been challenging, in large part owing to the difficulty of delivering the therapeutic gene to cells within the mass of muscle tissue. A recent study of the adeno-associated virus capsid provides a candidate vector for...
- N Engl J Med 2022; 386:1005-1007
Malaria continues to devastate people’s health and livelihoods worldwide. In October 2021, the WHO for the first time recommended the large-scale use of a malaria vaccine for children living in areas with moderate-to-high malaria transmission.
- N Engl J Med 2022; 386:988-989
Well before the Covid-19 pandemic disrupted tuberculosis care,1 long treatment duration has been a weak link in the continuum of care. But the past decade has been a turning point in the pioneering of shorter treatment and differentiated care, as opposed to the traditional, one-size-fits-all approach. Shortening of...
- N Engl J Med 2022; 386:911-922
Completing 6 months of treatment for tuberculosis in children is a challenge. In this open-label trial involving 1204 children in sub-Saharan Africa and India, 4 months of antituberculosis therapy was noninferior to 6 months of therapy.