Crysvita (burosumab-twza) / Ultragenyx, Kyowa Kirin - LARVOL DELTA

X-Linked Hypophosphatemia Management in Adults: An International Working Group Clinical Practice Guideline. (PubMed, J Clin Endocrinol Metab) - "In adults with XLH and fractures or pseudofractures, burosumab is recommended over no therapy (strong recommendation, GRADEd). Additionally, burosumab is suggested as the preferred treatment compared to conventional therapy (conditional recommendation, GRADEd) in the absence of fractures or pseudofractures. If burosumab is not available, symptomatic adults should be treated with conventional therapy (Non-GRADEd recommendation)."

Clinical guideline • Journal • Musculoskeletal Diseases • Orthopedics • Renal Disease • FGF23

Is Burosumab a Good Choice?: Experience in Adult X-Linked Hypophosphatemic Rickets in a Turkish Patient (ESPE-ESE 2025) - No abstract available

Clinical

Analysis of the therapeutic effect of Burosumab in the treatment of 6 children with X-Linked Hypophosphatemia (ESPE-ESE 2025) - No abstract available

Clinical • Renal Disease

The management of X-Linked Hypophosphatemia: Oral phosphate and calcitriol supplementation vs. Burosumab. A case report (ESPE-ESE 2025) - No abstract available

Case report • Clinical • Renal Disease

Successful Treatment of Autosomal Dominant Hypophosphatemic Rickets with Burosumab: A Case Report (ESPE-ESE 2025) - No abstract available

Case report • Clinical

Non-interventional Post-Authorisation Safety Study of Burosumab in the Treatment of Children and Adolescents with X-linked Hypophosphataemia: Second Interim Analysis (ESPE-ESE 2025) - No abstract available

Clinical • Renal Disease

Acral Mesenchymal Tumor Leading to Tumor-Induced Osteomalacia: Case Report and Literature Review. (PubMed, AACE Clin Case Rep) - "Emerging therapies, such as FGF-23 inhibitors like burosumab, provide alternatives for nonlocalizable or unresectable tumors. This case emphasizes the need for increased clinician awareness, multidisciplinary approaches, and advances in diagnostic imaging to reduce delays in diagnosing TIO. Further research is necessary to elucidate the pathophysiology, explore genetic associations, and improve treatment options for this debilitating condition."

Journal • Musculoskeletal Diseases • Oncology • Orthopedics • Renal Disease • FGF23

Tumor-induced rickets/osteomalacia (TIO): diagnostic pitfalls and therapeutic options. (PubMed, J Bone Miner Res) - "She was first treated with active vitamin D and phosphate salt followed by burosumab which alleviated her symptoms...Wide excision of the lesion corrected hypophosphatemia. The differential diagnoses and treatment of hypophosphatemic rickets/osteomalacia are discussed."

Journal • Oncology • Renal Disease • FGF23

Office Blood Pressure and Obesity in Children with X-Linked Hypophosphatemia. (PubMed, Calcif Tissue Int) - "Spearman rank correlation analysis revealed significant associations between both systolic (r = 0.24; p < 0.01) and diastolic (r = 0.20; p < 0.05) blood pressure with BMI, while the mode of treatment, i.e. burosumab versus phosphate supplements and active vitamin D, was no significant correlate. Children with XLH present with elevated office blood pressure values, associated with elevated BMI."

Journal • Observational data • Cardiovascular • Genetic Disorders • Hypertension • Obesity • Pediatrics • Pulmonary Arterial Hypertension • Renal Disease

Examining Burden of Disease and Access to Care in Adults with X-Linked Hypophosphatemia in Brazil (ISPOR 2025) - "Despite generally comparable measures of XLH disease burden, far fewer Brazilian adults received burosumab (5/50; 10%) versus the overall DMP population (332/481; 69%). This highlights the unmet need for access to adequate treatment for Brazilian adults with XLH, which likely stems from the lack of reimbursement in this country despite marketing authorization of burosumab 5 years ago."

Clinical • HEOR • Genetic Disorders • Pain • Renal Disease • FGF23

Methodology for the international working group clinical practice guidelines on X-linked hypophosphatemia in children and adults. (PubMed, J Bone Miner Metab) - "For these questions, the panelists and methodologists collaboratively framed PICO (Population, Intervention, Control, and Outcomes) questions and conducted four systematic reviews assessing the impact of medical therapy-using either burosumab or phosphate and active vitamin D-on patient-important outcomes in the XLH population as well as the impact of medical intervention compared to no treatment...Using a rigorous narrative literature review, the panel developed non-GRADEd recommendations including guidance for pregnant women, patients with dental complications, and other areas where evidence is limited. This article summarizes the methodology utilized for the development of both GRADEd and non-GRADEd recommendations for patients with XLH."

Clinical guideline • Journal • Renal Disease

Two Cases of Tumor-induced Osteomalacia Resulting in Surgical Resection During Burosumab Therapy. (PubMed, Intern Med) - "Despite tumor removal, the serum FGF23 levels remained elevated. In the postoperative monitoring of patients treated with burosumab, physicians should focus on the serum and urine levels of phosphate rather than FGF23."

Journal • Oncology • Renal Disease • FGF23

X-Linked Hypophosphataemia and Burosumab: A Systemic Disease With a New Treatment. (PubMed, J Paediatr Child Health) - "This review summarises what is known, and more importantly what is unknown, about burosumab use in X linked hypophosphataemia. We highlight important areas for future research to better understand the impact of burosumab in XLH, improve management of XLH, assess cost benefit of, and advocate for fair and equitable access to burosumab."

Journal • Review • Dental Disorders • Endocrine Disorders • Immunology • Metabolic Disorders • Musculoskeletal Diseases • Nephrology • Orthopedics • Osteoarthritis • Pain • Renal Disease • Rheumatology • FGF23

X-Linked Hypophosphatemia Management in Children: An International Working Group Clinical Practice Guideline. (PubMed, J Clin Endocrinol Metab) - "These clinical practice guidelines provide an update of the current evidence on the diagnosis and management of XLH and provide a comprehensive guidance for multidisciplinary healthcare professionals involved in the care of children with XLH."

Clinical guideline • Journal • Pediatrics • Renal Disease

Burosumab Treatment in a Girl With Cutaneous Skeletal Hypophosphatemia Syndrome: 2-Year Follow-Up. (PubMed, Am J Med Genet A) - "We report a girl with CSHS confirmed by somatic HRAS pathogenic variant. Conventional treatment was replaced by burosumab, with healing of bone rickets, and a clear improvement in mineral homeostasis and physical functioning after 2 years of treatment."

Journal • Endocrine Disorders • Pain • Rare Diseases • Renal Disease • FGF23 • HRAS • KRAS

Impact of burosumab on the quality of life of patient with X-linked hypophosphatemic rickets (XLH) aged 0-18 years at the Hospital Infantil de México Federico Gómez (HIMFG), Mexico City (IPNA 2025) - "All patients reported an improvement in the quality of life score by the sixth month in all areas examined using the PedsQL4.0 instrument (physical, emotional, social and school) with an initial median of 22.8 versus 62.0 at month 6 (p<0.05). Conclusions Our findings, despite a limited sample (N8), indicate that this new drug modifies the quality of life of XLH patients through appropriate prescription, adherence to treatment and frequent follow-up."

Clinical • HEOR • Genetic Disorders • Nephrology • Pediatrics

Experience of using 104 weeks of burosumab in patients with XLH in a tertiary center of Mexico at the Instituto Nacional de Pediatria (IPNA 2025) - "Conclusions ConclusionTreatment with Burosumab is a safe medication with a positive impact on patients regarding clinical and radiological improvement. We consider that FA can be used as a bone marker and response to treatment."

Clinical • Dental Disorders • Dermatology • Metabolic Disorders • Nephrology • Otorhinolaryngology • Pediatrics • Renal Disease • FGF23

Management of X-Linked and Other Genetic Hypophosphatemias in Algiers (IPNA 2025) - "No patient received Burosumab (not available).The majority of patients had a favourable evolution with shortening of the inter condylar distance in two patients after 6 months of treatment, decrease of alkaline phosphatases (upper limit of normal).However, only one patient experienced treatment-related side effects such as hypercalciuria, necessitating a reduction in the dose of 1-alpha hydroxyvitamin D, and only one patient was diagnosed with an associated malformation...Late diagnosis often precludes the possibility of improving bone damage. The aim of supplementation treatment is to reduce bone deformities while avoiding complications, in particular nephrocalcinosis, while awaiting specific treatment which is not available in our country."

Metabolic Disorders • Musculoskeletal Diseases • Musculoskeletal Pain • Nephrology • Orthopedics • Pain • Pediatrics • Renal Disease

Children with X-linked hypophosphatemia show elevated office blood pressure associated with body mass index and conventional therapy: An analysis of the German/ Swiss XLH Registry (IPNA 2025) - "Burosumab, a monoclonal anti-FGF23 antibody, was shown to be superior to conventional treatment in healing of rickets...Conclusions Children with XLH present with elevated office BP values which is associated with BMI and conventional therapy. Monitoring of cardiovascular health and its risk factors in children with XLH appears to be important."

Clinical • Cardiovascular • Hypertension • Metabolic Disorders • Nephrology • Obesity • Pulmonary Arterial Hypertension • Renal Disease • FGF23

Combined Treatment With Leuprolide Acetate and Burosumab in X-linked Hypophosphatemia and Precocious Puberty: A Therapeutic Response. (PubMed, AACE Clin Case Rep) - "Thus, CPP can be treated in patients with XLH, who may have compromised height outcomes, using synthetic gonadotropin-releasing hormone analogs. In the described XLH patient with CPP, the combined use of gonadotropin-releasing hormone analogs and burosumab was a safe strategy to stabilize pubertal progression and bone age, minimize anthropometric loss, and avoid exacerbating bone deformities."

Journal • Endocrine Disorders • Musculoskeletal Diseases • Orthopedics • Renal Disease

Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia (clinicaltrials.gov) - P2 | N=12 | Completed | Sponsor: National Institute of Dental and Craniofacial Research (NIDCR) | Active, not recruiting ➔ Completed | Trial completion date: Sep 2030 ➔ Nov 2024 | Trial primary completion date: Sep 2030 ➔ Nov 2024

Trial completion • Trial completion date • Trial primary completion date • Renal Disease

Treatment Advances in Tumor-Induced Osteomalacia. (PubMed, Calcif Tissue Int) - "Oral phosphate and calcitriol were the mainstay of medical therapy, however, the development of burosumab, a monoclonal blocking antibody to FGF23, has introduced an approved therapy that improves hypophosphatemia and symptoms in patients with TIO. In select cases, cinacalcet can be an effective adjuvant to phosphate and calcitriol...Infigratinib, a selective FGFR tyrosine-kinase inhibitor targeting a causative tumoral fusion protein, can reverse the biochemical findings of TIO and possibly reduce tumor mass; however, its use is constrained by serious side effects. Overall, innovations in medical and interventional treatments have broadened therapeutic options for patients with PMTs, particularly in cases where a curative surgical resection is not possible."

Journal • Review • Fatigue • Musculoskeletal Diseases • Musculoskeletal Pain • Oncology • Orthopedics • Pain • Renal Disease • FGF23 • FGFR

Clinical practice recommendations for the diagnosis and management of X-linked hypophosphataemia. (PubMed, Nat Rev Nephrol) - "However, the management of XLH has evolved in the past few years owing to the availability of burosumab, a fully humanized monoclonal antibody that neutralizes circulating FGF23. Here, we provide updated clinical practice recommendations for the diagnosis and management of XLH to improve outcomes and quality of life in these patients."

Journal • Review • Immunology • Musculoskeletal Diseases • Orthopedics • Osteoarthritis • Pain • Renal Disease • Rheumatology • FGF23

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Pediatric Patients with X-Linked Hypophosphatemia. (PubMed, J Clin Endocrinol Metab) - "Our review indicates that burosumab likely provides benefits to children by preventing lower limb deformity and improving physical health QoL while potentially increasing height. However, burosumab may also increase adverse events. Our review found limited evidence regarding the impact of conventional therapy compared to no treatment on final height. Further research is required to understand the long-term effect of medical therapy in children."

Journal • Dental Disorders • Pediatrics • Renal Disease

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia. (PubMed, J Clin Endocrinol Metab) - "No formal comparisons between burosumab and conventional therapy in adults exist. Evidence for conventional therapy versus no treatment is very uncertain. Our review highlights the need for more data on the long-term effects of burosumab and conventional therapy on patient-important outcomes in adult patients with XLH."

Journal • Review • Dental Disorders • Fatigue • Musculoskeletal Diseases • Orthopedics • Pain • Renal Disease