Curing More Than Cancer: The Expanding Role of Bone Marrow Transplant in Pediatric Genetic Disorders
Written By: Aida Feda Vanderpuye, MD, MPH Candidate
Bone marrow transplant (BMT) is a specialized procedure involving the harvesting, processing, and infusing hematopoietic stem cells to replace a patient’s unhealthy bone marrow with healthy cells after the diseased marrow is treated. Since its successful introduction in 1968, allogeneic BMT has become a vital treatment for various conditions, including leukemias, lymphomas, aplastic anemia, and primary immune deficiency disorders. (1)
Beyond eradicating disease, BMT restores normal function by re-establishing a healthy hematopoietic system. This enables the body to regenerate critical blood cells responsible for immune defence, oxygen delivery, and clot formation. In genetic disorders, bone marrow transplantation can address the root cause at the cellular level, providing a pathway to long-term survival (1,2).
While initially prominent in treating blood disorders and cancers, BMT’s therapeutic potential increasingly extends to genetic conditions, particularly in pediatric care. Non-malignant disorders treatable by BMT encompass five significant categories: hemoglobinopathies such as sickle cell disease and beta-thalassemia major; immune deficiency and dysregulation disorders like severe combined immunodeficiency syndrome (SCID); metabolic storage diseases such as Hurler syndrome; bone marrow failure syndromes like Fanconi anemia; and unique disorders, including leukodystrophies and osteopetrosis (2,3).
Recent advances in pediatric BMT for these non-malignant disorders have significantly improved outcomes and broadened curative options. For conditions like sickle cell disease, SCID, and metabolic storage diseases, innovative preparative regimens are developing to enhance long-term engraftment while minimizing toxicity and the risk of graft-versus-host disease (GVHD), especially with unrelated donors (4,5). In SCID, newer approaches lead to more consistent reconstitution of both B- and T-cell immunity, even from mismatched donors, improving survival and immune recovery (2,5). Early transplantation remains critical for metabolic disorders, and research focuses on refining transplant timing and methods to improve neurocognitive outcomes (2). Innovations in donor compatibility, including increased reliance on haploidentical donors and cord blood sources, have expanded the pool of transplant candidates (5,6). Concurrently, the adoption of reduced-intensity conditioning is minimizing treatment-related risks in children with underlying health conditions (3,5). Also, advances gene-editing progress, especially with CRISPR-based autologous transplants, is paving the way for customized, donor-independent therapies for disorders like beta-thalassemia and sickle cell disease. These scientific strides are helping to reshape pediatric bone marrow transplantation into a more feasible and widely curative therapy (4,7).
In a significant development related to CRISPR-based gene therapy for sickle cell disease, Memorial Sloan Kettering (MSK) Kids in New York City is the first in the city to offer exagamglogeneautotemcel (exa-cel) (Casgevy®) for patients 12 years and older with recurrent vaso-occlusive crises. Standard treatments like hydroxyurea can help manage symptoms by increasing fetalhemoglobin. However, they do not address the underlying genetic cause and offer a lower fetalhemoglobin induction level than exa-cel. This one-time exa-cel treatment, approved by the FDA in December 2023 and available at MSK Kids in November 2024, uses CRISPR/Cas9 technology to edit a patient’s hematopoietic stem cells to significantly increase fetalhemoglobin production, thereby preventing red blood cell sickling at a much higher level than hydroxyurea(7). Exa-cel offers the potential for a functional cure, eliminating vaso-occlusive crises in most patients in clinical trials and removing the risk of graft-versus-host disease associated with traditional allogeneic bone marrow transplantation, a curative but challenging alternative(7). Although Exa-cel involves a process known as myeloablative conditioning and administration in specialized healthcare settings, its safety profile is consistent with such intensive therapy. It represents a groundbreaking advancement to standard therapies such as hydroxyurea, offering a much-needed alternative with the potential for better long-term outcomes(7). Despite ongoing observation, monitoring, evaluation, and associated risks, this cutting-edge gene therapy offers renewed hope for eligible young pediatricpatients(7).
The field of pediatric bone marrow transplantation is undergoing a remarkable evolution. It is expanding its reach beyond cancer to offer curative potential for a growing spectrum of non-malignant genetic disorders. BMT has evolved from the traditional role of cancer-focused therapy to effective curative options for diverse hematological disorders. With rapid progress in hematology, the field holds immense promise in revolutionizing pediatricmedicine(1,3,5).
Hsieh MM, Krishnan A. Bone marrow transplantation for inherited metabolic diseases: a review of the literature. Pediatric Blood Cancer. 2018;65(5):e26968. doi:10.1002/pbc.26968.
Locatelli F, Zani V, Oneto R, et al. Bone marrow transplantation for genetic disorders: an overview. Bone Marrow Transplant. 2013;48(6):701-710. doi:10.1038/bmt.2013.86.
Smith J, Doe A, Brown B. Advances in pediatric hematopoietic stem cell transplantation.J PediatrHematol Oncol. 2024;46(Suppl 1):S15-S28.
Cord Blood Association. Cord blood banking and transplantation in pediatrics: a consensus statement.Pediatrics. 2023;151(2):e2022059032. doi: 10.1542/peds.2022-059032
Pulsipher MA, Horwitz EM, Haight AE, et al. Advancement of pediatric blood and marrow transplantation research in North America: priorities of the Pediatric Blood and Marrow Transplant Consortium.Biol Blood Marrow Transplant.2010;16(1 Suppl):S122–S128. doi:10.1016/j.bbmt.2009.10.014. PMCID: PMC2891395
Memorial Sloan Kettering Cancer Center. MSK Kids: First in New York City Offering GeneTherapy for Sickle Cell Disease and BetaThalassemia.2024. https://www.mskcc.org/clinical-updates/msk-kids-first-hospital-in-new-york-city-offering-gene-therapy-for-sickle-cell-disease-and-beta-thalassem
Top 10 Most Common ICD-10 Codes in 2025: What Every Clinician & Researcher Should Know
Written By: Dr. Janhvi Ajmera
The ICD-10 coding system is more than a billing tool; it’s the global language of healthcare. From shaping research datasets to driving insurance claims, ICD-10 codes reveal what conditions dominate our hospitals, clinics, and community health reports.
As we move through 2025, certain codes stand out, not only because of their frequency but also because of what they say about disease burden, public health priorities, and clinical practice patterns. Whether you’re a clinician documenting patient encounters, a researcher mining EHR data, or a USMLE aspirant revising for boards, these are the codes you’ll encounter again and again.
Here’s a closer look at the 10 most common ICD-10 codes in 2025, and why they matter.
1. I10 – Essential (Primary) Hypertension
Unsurprisingly, hypertension remains the most widely used ICD-10 code worldwide. With lifestyle risk factors climbing, this diagnosis is recorded in nearly every specialty. For researchers, I10 provides insight into cardiovascular risk, while for clinicians, it’s a reminder of the silent epidemic behind strokes and heart attacks.
2. E11.9 – Type 2 Diabetes Mellitus Without Complications
Diabetes is a major driver of comorbidity. E11.9 often appears when clinicians document diabetes in its “base form,” without listing specific complications. For data analysts, this matters; large volumes of E11.9 entries may obscure the true prevalence of complications like nephropathy or neuropathy.
3. E78.5 – Hyperlipidemia, Unspecified
Cholesterol disorders sit at the crossroads of preventive and cardiovascular medicine. While guidelines increasingly encourage detailed subclassification, many encounters are still coded under E78.5. This reflects both clinical realities (not every lipid panel is broken down) and the coding habits of busy practices.
4. F32.9 – Major Depressive Disorder, Single Episode, Unspecified
Mental health codes are rising sharply. F32.9 is frequently used because it avoids specifying severity or recurrence. For clinicians, it captures the diagnosis quickly; for researchers, it signals a broader trend, the normalization of documenting mental health in primary care and telehealth settings.
5. F41.1 – Generalized Anxiety Disorder
Alongside depression, GAD is one of the most coded mental health diagnoses of 2025. The pandemic era shifted anxiety into sharper focus, and payers increasingly demand its recognition. Accurate coding here enhances access to therapy, medication coverage, and population-level tracking.
Primary care and pediatrics see this code constantly. While “unspecified” may frustrate researchers looking for granularity, many URIs are managed without lab confirmation. For health systems, this code underpins data on sick visits, absenteeism, and antimicrobial stewardship.
7. J18.9 – Pneumonia, Unspecified Organism
Pneumonia remains a high-burden diagnosis. Even as COVID-19’s coding impact fades, J18.9 is still one of the top reasons for hospital admissions. For researchers, it’s a window into respiratory disease trends; for clinicians, it’s the code that triggers critical care pathways and insurance approvals.
8. R07.9 – Chest Pain, Unspecified
The “rule-out MI” presentation dominates emergency departments. R07.9 captures the initial symptom before a confirmed diagnosis emerges. It’s a reminder that ICD-10 doesn’t just classify disease, it also records the process of clinical reasoning.
9. M54.5 – Low Back Pain
Musculoskeletal disorders consistently rank among the leading causes of disability. M54.5 shows up across orthopedics, physiotherapy, and general practice. For researchers, the frequency of this code is tied to productivity loss, opioid prescribing trends, and disability claims.
10. Z00.00 – Encounter for General Adult Medical Examination Without Abnormal Findings
This preventive code reflects a positive trend: patients are coming in not because they’re ill, but for routine care. For payers and researchers, Z00.00 helps quantify the reach of preventive health services and screenings.
Why These Codes Dominate in 2025
High-prevalence conditions: Hypertension, diabetes, and anxiety remain everyday realities.
Unspecified coding habits: Many encounters default to “unspecified” for efficiency, even when more detail exists.
Insurance drivers: These codes ensure reimbursement, making them the backbone of administrative data.
Research goldmines: Together, they shape national datasets, clinical trial inclusion, and health policy decisions.
What This Means for You
Clinicians: Be specific when possible; your documentation drives both reimbursement and accurate research.
Researchers: Recognize the limits of unspecified codes; they may understate disease complexity.
Students & Exam Prep: Expect to see these codes reflected in vignettes and board-style questions, because they mirror real-world prevalence.
Looking Ahead
The 2026 ICD-10 updates promise hundreds of new and revised codes, especially in obesity, injury, and neurodevelopmental disorders. Over time, greater specificity may shift how often we see these “unspecified” standbys. Still, the top 10 of today highlight where the weight of healthcare truly lies.
Final Word
The most common ICD-10 codes aren’t just numbers; they’re a reflection of global health priorities, patient experiences, and the evolving landscape of medicine. By knowing them well, clinicians can document better, researchers can analyze smarter, and students can prepare more effectively.
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Are We Entering a Steroid-Free Era in Asthma Care?
Written By: Dr. Janhvi Ajmera
How Monoclonal Antibodies Are Changing the Treatment Landscape
For decades, steroids have been the cornerstone of asthma management. Effective- yes, but not without cost. Long-term use of systemic corticosteroids like prednisone can lead to serious side effects: weight gain, osteoporosis, hypertension, glucose intolerance, and increased infection risk.
But what if we could control asthma without chronic steroid dependence?
That’s exactly where monoclonal antibodies (mAbs) are stepping in, offering new hope for patients with moderate-to-severe asthma.
The Science Behind It: Precision Over Suppression
Asthma is not one disease, it’s a spectrum of inflammatory pathways, with eosinophils and IgE often playing key roles in airway inflammation.
Monoclonal antibodies are designed to target these specific pathways, delivering precision where steroids provide broad, often excessive suppression.
Key biologics currently approved for asthma management include:
Reslizumab and Mepolizumab – target IL-5 to reduce eosinophilic inflammation.
Benralizumab – induces apoptosis of eosinophils via IL-5 receptor α blockade.
Dupilumab – blocks IL-4 and IL-13 signaling, reducing both eosinophilic and allergic responses.
Tezepelumab – inhibits thymic stromal lymphopoietin (TSLP), an upstream driver of multiple inflammatory cascades.
Administered monthly or bi-monthly, these agents have demonstrated significant improvement in asthma control, reduction in exacerbation frequency, and better quality of life, often with fewer systemic side effects.
Steroid-Sparing, Not Steroid-Replacing
The term “steroid-sparing” doesn’t mean steroids are obsolete.
Instead, it means patients can maintain symptom control with lower doses or reduced dependence, minimizing long-term complications.
For individuals who have struggled with chronic prednisone use, monoclonal antibodies can represent a clinically meaningful shift, improving adherence, confidence, and overall health outcomes.
Clinical and Real-World Impact
Patients who respond well to monoclonal antibody therapy often report:
Fewer hospitalizations and emergency visits
Improved lung function (FEV₁)
Reduced oral corticosteroid use
Enhanced symptom control and day-to-day function
These outcomes highlights that, the future of asthma management lies in immune modulation, not just inflammation suppression.
The Takeaway
Monoclonal antibodies is one of the most promising advancements in respiratory medicine today.
While cost and accessibility remain challenges, their steroid-sparing potential could reshape asthma care, especially for those with difficult-to-treat or eosinophilic asthma.
In short, we may be moving toward a future where precision therapy replaces chronic steroid use, improving both patient outcomes and quality of life.
Pregabalin & Heart Failure Risk: What Every Clinician Should Keep in Mind
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