Children undergoing hematopoietic cell transplantation (HCT) for nonmalignant disorders are increasingly surviving long-term, shifting the focus from procedural success to durable immune recovery, infection prevention, revaccination, organ health, development, and quality of life. General pediatricians play a central role in shared care after HCT, as children transition from transplant-centered monitoring back to community-based care. Post-HCT immune recovery is heterogeneous and depends on diagnosis, donor source, graft source, conditioning, serotherapy exposure, graft-versus-host disease (GVHD), immunosuppression, and prior infections. Infection risk persists beyond neutro-phil recovery and may be prolonged in children with delayed T-cell or B-cell recovery, chronic GVHD, or ongoing immunosuppression. Revaccination is essential, as pretrans-plant immunity is often lost. This review provides a practical framework for pediatricians caring for children after nonmalignant HCT, with emphasis on infection prevention, immunization, survivorship monitoring, and the emerging role of data-driven tools to support individualized follow-up.
The landscape of pediatric hematopoietic cell transplantation for nonmalignant disorders has evolved through disease-adapted conditioning, pharmacokinetic-guided dosing, expanded donor options, and gene therapy (GT). These advances have transformed curative strategies for children with select inborn errors of immunity, hemophagocytic lymphohistiocytosis, hemoglobinopathies, bone marrow failure syndromes, and select metabolic disorders. Early diagnosis, especially newborn screening for severe combined immunodeficiency, enables timely intervention, with contemporary series reporting more than 90% survival in select early-treated infants. Similarly, modern matched related donor transplantation for pediatric severe aplastic anemia approaches 98% 3-year survival. Fludarabine-based platforms combined with busulfan, melphalan, or treosulfan support durable engraftment with reduced toxicity, while matched unrelated, haploidentical, and cord blood donors expand access. GT now offers a parallel curative platform for select sickle cell disease, transfusion-dependent beta-thalassemia, and immunodeficiencies. For pediatricians, early referral before irreversible complications is critical. This review proposes a five-axis framework to guide referral, treatment selection, and survivorship planning.
Hemoglobinopathies are inherited disorders of hemoglobin structure or production. Sickle cell disease (SCD) results from a beta-globin mutation causing hemoglobin polymerization, red blood cell sickling, vaso-occlusion, and hemolytic anemia. Thalassemia results from reduced or absent alpha- or beta-globin synthesis, ranging from asymptomatic carrier states to transfusion-dependent anemia. This article summarizes the pathophysiology, diagnosis, management, and curative options for SCD and transfusion-dependent thalassemia, emphasizing allogeneic hematopoietic stem cell transplantation and approved gene therapies. We discuss indications, counseling, barriers to access, toxicity, donor availability, fertility counseling, and comparative features of cellular therapies. Advances in transplantation, gene addition, and gene editing may expand access to curative-intent therapy and improve outcomes for selected patients.
Primary immunodeficiencies, now more broadly termed as "inborn errors of immunity (IEI)," are rare genetic disorders associated with recurrent infections, immune dysregulation, autoimmunity, lymphoproliferation, and malignancy risk. Early recognition and diagnosis are essential to reduce infection-related morbidity, prevent organ damage, and identify children who may benefit from curative therapy. A comprehensive history, physical examination, targeted immune evaluation, and genetic testing can support diagnosis, while early supportive measures may reduce infection risk before definitive therapy. Treatment may include antimicrobial prophylaxis, immunoglobulin replacement, enzyme replacement, immune-modifying agents, gene-based therapies, and allogeneic hematopoietic cell transplantation (HCT), which can be curative for select IEI. However, HCT requires careful pretransplant evaluation, as outcomes are influenced by disease biology, infection burden, organ function, donor availability, graft-versus-host disease risk, and conditioning toxicity. This review focuses on recognition of IEI, initial evaluation, supportive care, and pre-HCT management for pediatricians.
Infantile hemangioma (IH) is a benign tumor that appears in the first year of life and typically involutes within 3 to 4 years. Ulcerative IH, the most common complication, poses a significant burden to patients. Early referral of high-risk cases reduces morbidity. Current literature on risk factors and prognosis is limited. Segmental and mixed IH in urogenital, head, neck, and lip areas show higher ulceration risk. Diagnosis is typically clinical; histopathology is rarely performed. Atypical cases require positive glucose transporter-1 immunohistochemistry. Larger lesions, delayed intervention, and bacterial infection worsen prognosis, prolong healing, and increase recurrence. Condition-dependent treatments, including oral propranolol (starting at 1 mg/kg or less daily, maintenance at 2 to 3 mg/kg daily), timolol prophylaxis, and wound management care combined with laser therapy, shorten therapy duration and minimize recurrence. Empiric antibiotics are indicated for infected ulcerative IH. Surgery is indicated for life-threatening cases (eg, chest wall hemangiomas requiring transfusion or airway obstruction) and high-morbidity cases (eg, lip lesions affecting speech or periorbital lesions impairing vision). The Infantile Hemangioma Referral Score helps clinicians identify high-risk patients who require prompt referral, thereby preventing delayed treatment.
Eating disorders (EDs) are prevalent in the adolescent and young adult populations, and there is a significant burden of disease with high morbidity and mortality. Many patients do not initially seek ED subspecialty care, and primary care clinicians must identify and treat these patients with the help of a multidisciplinary team of ED-specialized dietitians, therapists, and psychiatrists. The primary care clinician must also be able to monitor for and identify medical instability or failure of outpatient treatment and recommend a higher level of care if necessary. Higher levels of care include medical inpatient treatment, psychiatric inpatient treatment, residential programs, partial hospital programs, and intensive outpatient programs. For optimal treatment outcomes, it is critical to select the appropriate level of care for each patient and step down as indicated.
Current obesity guidelines from the American Academy of Pediatrics include earlier treatment with pharmacotherapy and bariatric surgery, when indicated, rather than the staged approaches used in the past. However, it is important to consider the implications this may have on adolescent patients at high risk of developing an eating disorder (ED). Therefore, clinicians treating patients with obesity encounter a challenge to broach the topic of obesity treatment tactfully to avoid inciting or exacerbating an underlying ED, while also following evidence-based guidelines for the treatment of obesity. This review will highlight the current treatments for overweight and obesity; review the importance of screening before, during, and after treatment; review weight stigma; help guide the clinician in assessing for EDs; and discuss areas for future research.
Substance use in adolescents is an important public health topic. It can affect cognitive development and is associated with negative outcomes, such as school truancy, low graduation rates, and increased high-risk behaviors (eg, unprotected sex, dangerous driving), as well as high rates of suicide, substance abuse, dependence in adulthood, and death by overdose. Substance use among adolescents declined during the coronavirus 2019 pandemic and has continued to do so, resulting in the lowest rates of substance use in decades. Despite this decrease, physicians and nonphysician practitioners still need to be prepared to navigate this issue in teenagers and young adults. The role of primary care physicians and other practitioners includes promotion of abstinence from substances, using validated screening questionnaires, and treatment through motivational interviewing and harm reduction, as well as referring to specialists, higher levels of care, and rehabilitation centers as needed.
Menstrual health is a key indicator of adolescent health and should be evaluated at routine visits using a patient-centered approach. It is important to understand normal menstrual patterns in order to better recognize when a presentation may be abnormal or concerning. Clinicians may frequently encounter challenges, particularly when caring for adolescents with disabilities. In this article, we review typical menstrual development and evaluation and management of abnormal uterine bleeding including amenorrhea, dysmenorrhea, and heavy menstrual bleeding. We also highlight special considerations for adolescents who are differently abled.
Adolescents increasingly construct aspects of their identity and form relationships by interacting on social media; yet, clinical frameworks lag behind technological change. This review synthesizes recent evidence on how platform design, usage patterns, and individual differences influence both benefits and harms. Problematic use is associated with sleep disturbance, mood and attention disorders, body image concerns, cyberbul-lying, and exposure to self-harm content, with disproportionate burdens for female patients and youth who are marginalized. Meanwhile, online communities can foster connection, coping, and help seeking, particularly for adolescents facing stigma or isolation. We propose a structured, developmentally informed workflow incorporating brief screening, targeted use of validated tools, and collaborative family media planning. Rather than centering total screen time, we emphasize function-focused, harm-reduction approaches to harness digital platforms as a tool for adolescent health.
Speech and language development are critical areas of a child's health and are monitored by primary care pediatricians at all routine visits. Given the prevalence of bilingual children and families in the United States, pediatric clinicians must be able to provide appropriate anticipatory guidance regarding bilingual language development in children. Pediatric clinicians must also be able to recognize signs of speech/language delays or disorders in bilingual children to provide appropriate evaluation and resources. This column aims to provide a foundation for pediatric clinicians in principles of bilingual language development and to address several common questions or misconceptions regarding bilingual language development that may be held by families, clinicians, and/or educators.
A growing body of research demonstrates that equity and diversity in pediatric medical education programs are imperative to strengthen the pediatric workforce and improve pediatric health outcomes. Despite this, there is limited actionable guidance for clinicians and educators on methods to mitigate inequities within the recruitment process. This narrative review summarizes evidence-based strategies to reduce disparities in the undergraduate and graduate medical education applicant selection process, including holistic review, data-driven selection, implicit bias training, reduction of structural barriers, and showcasing existing diversity within programs. The review concludes with calls to action for leaders of programs and professional societies to pursue recruitment practices that are fair and evidence-based, as well as to create a more representative workforce. Additionally, recruitment improvements are only part of the puzzle, and programs must ensure an equitable and inclusive learning environment for diverse learners to have the best opportunity for success.
Children in immigrant families (CIF) represent one in four children in the United States and face disproportionate health risks shaped by immigration policy, structural racism, trauma, and barriers to care. Using a socioecological framework, this article reviews the current state of pediatric immigrant health and outlines opportunities for pediatricians to advance equity at the individual, organizational, and structural levels. Clinicians can follow expert clinical care guidelines, practice trauma-informed care and safe documentation practices, and partner with qualified medical interpreters. Health systems can create safe spaces for immigrant families and collaborate with researchers to promote community-led cohort building while protecting patients from identification. Finally, pediatricians play a vital role in advocating for inclusive insurance coverage and equitable health policies. Advancing care for CIF requires coordinated efforts across all levels of influence to ensure that every child, regardless of birthplace or legal status, receives care grounded in dignity, trust, and justice.
Migraine variants in childhood present with diverse and often nonheadache symptoms, complicating diagnosis and management. This review aims to clarify the clinical features, differential diagnoses, and therapeutic strategies for pediatric migraine variants and precursors, including infantile colic, benign paroxysmal torticollis, recurrent vertigo, cyclic vomiting syndrome, abdominal migraine, acute confusional migraine, and Alice in Wonderland syndrome. A narrative approach was used, integrating case-based discussion and current diagnostic criteria. Key findings highlight the paroxysmal nature of these syndromes, frequent family history of migraine, and the importance of excluding other causes. Most migraine variants are self-limited and benefit from supportive care, trigger avoidance, and age-appropriate pharmacologic prophylaxis when indicated. Early recognition and reassurance are essential in preventing unnecessary investigations and improving outcomes. This review underscores the need for clinician awareness and individualized management to optimize care for children with migraine variants.
