hydroxyurea

Ongoing treatment with hydroxyurea was substituted with ropeg (week 0: 250 mcg; week 2: 350 mcg; week 4 onwards: 500 mcg every 2 weeks). In conclusion, ropeg was safe and induced CHCR associated with significant molecular responses in patients with early MF. ClinicalTrials.gov Identifier: NCT04988815.

All TKIs are not recommended during the first trimester due to their risk of teratogenesis, but imatinib and nilotinib may be cautiously used from Weeks 16-18 onward. Non-TKI therapies, such as hydroxyurea and interferon-α, are considered safe throughout pregnancy. Data on ponatinib and asciminib remain insufficient to allow the safe use of these agents during pregnancy. Future research should aim to improve treatment-free remission rates through novel agents and combination strategies to allow a higher proportion of younger patients to discontinue therapy. Clinicians should always counsel women on pregnancy risks during therapy while reassuring male patients of TKI safety when fathering children.

These results reveal a relatively homogeneous epidemiological profile across the Latin American region and underscore the need for more multicenter studies to better characterize pH- MPNs in Ecuador and the region, to optimize diagnostic and treatment strategies.

P2, N=70, Not yet recruiting, Uma Borate | Initiation date: Jan 2026 --> Apr 2026

P4, N=76, Terminated, Novartis Pharmaceuticals | Trial completion date: Jan 2027 --> Feb 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Jan 2027 --> Feb 2026; Sponsor Decision

P1, N=25, Terminated, Imugene Limited | N=50 --> 25 | Trial completion date: May 2029 --> Feb 2026 | Active, not recruiting --> Terminated | Trial primary completion date: Jun 2027 --> Feb 2026; Portfolio prioritization

P1, N=30, Recruiting, University of Chicago | Trial completion date: Jan 2026 --> Jan 2028 | Trial primary completion date: Jan 2026 --> Jan 2028

P2, N=60, Recruiting, Chengdu Zenitar Biomedical Technology Co., Ltd | Active, not recruiting --> Recruiting

Hollow mesoporous Prussian blue (HMPB) nanozymes coloaded with hydroxyurea (HU) and thapsigargin (TG) are embedded within a poly(γ-glutamic acid) microneedle matrix, allowing localized and minimally invasive intratumoral delivery. This trimodal self-amplifying immune cascade enhances tumor immunogenicity, remodels the tumor immune microenvironment, and elicits potent local and systemic T cell-mediated antitumor responses. Collectively, this work establishes a nanozyme-microneedle-integrated strategy for the precise orchestration of innate and adaptive antitumor immunity.

The patient was stabilised with hydroxyurea and subsequently commenced on imatinib, achieving haematological remission, while supportive measures addressed the multisystem complications of MPS VI. This case highlights the need for vigilance in recognising malignancy in children with rare genetic disorders, the limitations of confirmatory testing in low-resource settings and the importance of multidisciplinary, individualised care when treatments for one condition may complicate the other.

P1/2, N=36, Not yet recruiting, Oryzon Genomics S.A.

P4, N=50, Recruiting, Emory University | Not yet recruiting --> Recruiting | Initiation date: Dec 2025 --> Apr 2026