Le attività di Cure2Children negli utlimi 5 anni

Negli ultimi cinque anni la Fondazione Cure2Children ha sviluppato e ampliato in modo significativo le proprie attività, rafforzando sia la cura diretta dei bambini affetti da malattie gravi sia le competenze dei sistemi sanitari locali nei paesi a risorse limitate. Grazie alla collaborazione consolidata con organizzazioni internazionali come DKMS
(Germania) e Sankalp India Foundation (India), il numero di bambini con malattie mortali curati ogni anno per patologie potenzialmente mortali è più che quintuplicato,
passando da meno di 50 a oltre 300.
Questa attività rappresenta oggi la più ampia esperienza internazionale nel campo delle emoglobinopatie – in particolare talassemia e anemia falciforme – tra le malattie non infettive pediatriche più diffuse e letali a livello globale. Ciò consente a Cure2Children di rispondere a una quota crescente delle richieste di aiuto che arrivano quotidianamente, soprattutto da Asia e Africa.
L’esperienza clinica accumulata ha inoltre permesso alla Fondazione di partecipare ai processi di accreditamento internazionale di massimo livello per i centri trapianto (FACT- JACIE) e di incrementare in modo sostanziale la produzione scientifica.
Nel dicembre 2025 si è conclusa la seconda edizione del corso European Society for Blood and Marrow Transplantation (EBMT) – programma EMPACT2 – dedicato alla
formazione avanzata in cure pediatriche nei Paesi a medio reddito, con particolare attenzione alla specializzazione del personale infermieristico.
Parallelamente, Cure2Children ha sostenuto l’avvio e la formazione di nuovi centri sanitari in India, Vietnam, Uzbekistan, Iraq, Nigeria, Uganda e Camerun, promuovendo
modelli di cura sostenibili e autonomi. Fondamentale anche l’attività di tipizzazione HLA gratuita, realizzata in collaborazione con DKMS, per facilitare l’accesso al trapianto di cellule staminali ematopoietiche nei
bambini privi di donatore compatibile in famiglia.
Pubblicazioni scientifiche
21. Nguyen HTK, Faulkner L, Bui SBB, Lederman HM, Ho NTT, Dang TT. Diffuse alveolar hemorrhage in a child with mild hemophilia A who underwent bone marrow transplantation for thalassemia: a case report. Front Hematol 2025; 4. doi:10.3389/frhem.2025.1657903.
22. Sevanthini BR, Hegde S, Sridhar T, Trivedi D, Reddy M, Shah V, Panchamia VM, Geetha T, Vora D, Pushpa H, Agarwal RK, Faulkner L. Short-term risks and benefits of hydroxyurea (hydroxycarbamide)- induced transfusion independence in patients with beta-thalassaemia syndromes. Br J Haematol. 2025 Aug 29. doi: 10.1111/bjh.70109. Epub ahead of print. PMID: 40884002. https://doi.org/10.1111/bjh.70109
23. Agarwal RK, Periyavan S, Trivedi D, Shah V, Reddy M, Mani G, Sedai A, Kumari A, Faulkner L. Measuring success of targeted screening and prevention for hemoglobinopathies. Front Public Health 2025; 13. doi:10.3389/fpubh.2025.1587738.
24. Agarwal RK, Dhanya R, Parmar L, Sedai A, Mani G, Dhar A, Hegde S, Periyavan S, Erappa N, S A, Kumar S, Rengaraj B, G N M, N M, Prasad RN, Fernandes R, Makkam A, Faulkner L. Prenatal hemoglobinopathy screening & prevention in India: A cross-sectional study. Indian J Med Res. 2025 May;161(5):441-448. doi: 10.25259/IJMR_1003_2024. PMID: 40844095. https://doi.org/10.25259/ijmr_1003_2024
25. Agarwal RK, Dhanya R, Trivedi D, Shah V, Reddy M, Marwah P, Sedai A, Ankita K, Parmar L, Faulkner L. Granulocyte Colony-Stimulating Factor-Primed Bone Marrow Transplantation Experience in 350 Matched Sibling Donor Grafts for Severe Thalassemia. Transplant Cell Ther. 2025 May;31(5):319.e1- 319.e8. Epub 2025 Jan 30. PMID: 39890022., https://doi.org/10.1016/j.jtct.2025.01.890.
26. Periyavan S, Kumar S, Mamatha, GN, Hegde S, Jain S, Dhanya R, Agarwal,RK, Faulkner L. HPLC First Approach in Detecting Thalassemia and Other Common Hemoglobinopathies Is More Cost and Time Effective. Front. Hematol. 2025, 4. https://doi.org/10.3389/frhem.2025.1461498.
27. Kandy ZAK, Nivargi S, Chaudhary B, Ramzan M, Abdullah F, Aboobacker F, Khalil C, Porta F, Faulkner L, Alshamshi H. Outcomes of Hematopoietic Stem Cell Transplantation (HSCT) From Matched Related Donors in Pediatric Patients: Insights From a Single Center in the United Arab Emirates. Cureus 2025; 17. doi:10.7759/cureus.89239.
28. Srivastava A, Duarte FB, Faulkner L. Feasibility and Experiences of HCT in Resource-Constrained Settings. In: Sureda A, Corbacioglu S, Greco R, Kröger N, Carreras E, editors. The EBMT Handbook: Hematopoietic Cell Transplantation and Cellular Therapies [Internet]. Cham: Springer International Publishing; 2024 [cited 2024 Apr 13]. p. 607–14. Available from: https://doi.org/10.1007/978-3-031- 44080-9_69,
29. Agarwal RK, Dhanya R, Sedai A, Ankita K, Parmar L, Ramprakash DS, Sandeep, Trivedi D, Shah V, Bhat N, and Faulkner L: Bone Marrow Quality Index: A predictor of acute GVHD in hematopoietic stem cell transplantation for thalassemia. Transplantation and Cellular Therapy, 2023; https://doi.org/10.1016/j.jtct.2023.07.014.
30. Agarwal N, Jothi K, Gambhir S, Rall G, Landwehr R, Faulkner L, Jagannathan L, Paul P, and Schmidt A: The DKMS-BMST Thalassemia program: Description, scope and impact. Pediatric Hematology Oncology Journal 2023. https://doi.org/10.1016/j.phoj.2023.07.001
31. Verna M, Canesi M, Conter V, Faulkner L Rovelli MA, Silvestri D, Majolino I, Biondi A, Chra Nawfal A, Faeq Mohammed V. Hematopoietic stem cell transplant start-up in Iraqi Kurdistan: results on thalassemia patients and analysis of the methodology. Transplantation and Cellular Therapy. 2023, https://doi.org/10.1016/j.jtct.2023.01.018.
32. Matthay K., Hylton J., Penumarthy N.,Khattab M., Soh S.Y., Nguyen H.T.K., Alcasabas A.P., Fawzy M., Saab R., Khan M.S., Ghandour K., Chantada G., Parikh N.S., Faulkner L., Lam C.G., and Howard S.C. ‘Global Neuroblastoma Network: An International Multidisciplinary Neuroblastoma Tumor Board for Resource-Limited Countries’. Pediatric Blood & Cancer, January 2022. https://doi.org/10.1002/pbc.29568.

33. Marwah P., Ramprakash S., Prasad S..TR., Mane Gizhlaryan M., Trivedi D., Shah V., Chitaliya A., Elizabeth S., Agarwal R.K., Dhanya R. and Faulkner L. ‘Is It Safe and Efficacious to Remove Central Lines in Pediatric Bone Marrow Transplant Patients with Platelets Less than 20,000/ml?’ EJHaem, January 2022. https://doi.org/10.1002/jha2.379.
34. Dhanya R., Agarwal R.K., Ramprakash S., Trivedi D., Shah V., Bhat N., Reddy M., Elizabeth S., Batool A., Khalid S. and Faulkner L. ‘Do Weekly Surveillance Cultures Contribute to Antibiotic Stewardship and Correlate with Outcome of HSCT in Children? A Multicenter Real-World Experience of 5 Years from the Indian Subcontinent’. Transplantation and Cellular Therapy, December 2021.https://doi.org/10.1016/j.jtct.2021.12.008.
35. Diaz-de-Heredia C., Bresters D., Faulkner L., Yesilipek A., Strahm B., Miano M., Dalle,J.-H., Peffault de Latour R., Corbacioglu S., 2021. Recommendations on hematopoietic stem cell transplantation for patients with Diamond–Blackfan anemia. On behalf of the Pediatric Diseases and Severe Aplastic
Anemia Working Parties of the EBMT. Bone Marrow Transplantation 31 August 2021. https://doi.org/10.1038/s41409-021-01449-w
36. Mehta P., Ramprakash S., Raghuram C.P., Trivedi D., Dhanya R-, Agarwal R.K., and Faulkner L. Pre- Transplant Donor-Type Red Cell Transfusion Is a Safe and Effective Strategy to Reduce Isohemagglutinin Titers and Prevent Donor Marrow Infusion Reactions in Major ABO-Mismatched Transplants’. Annals of Hematology, 19 June 2021. https://doi.org/10.1007/s00277-021-04571-3.
37. El Fakih R. , Greinix H., Koh M., …Faulkner L., … Aljurf M. Worldwide Network for Blood and Marrow Transplantation (WBMT) Recommendations Regarding Essential Medications Required to Establish an Early Stage Hematopoietic Cell Transplantation Program. Transplantation and Cellular Therapy, 21 dicembre 2020. https://doi.org/10.1016/j.jtct.2020.12.015.
38. de la Fuente J., Gluckman E., Makani J., Telfer P., Faulkner L., Corbacioglu S., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J., Hereda Diaz C., Feuchtinger T., Locatelli F., Lucchini G., Galimard J., Gonzalez Vincent M., Handgretinger R., Kleinschmidt K., Lawitschka A., Perez Martinez A., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., Yesilipek A. The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene
editing- The Lancet Haematology, 7 (12):e902-e911, 2020. https://doi.org/10.1016/S2352- 3026(20)30283-0
39. Ramprakash S., Agarwal R., Dhanya R., Marwah P., Soni R., Yaqub N., Fatima I., Gooneratne L., Williams S., Khalid S., Sen S. and Faulkner L.: Splenomegaly may increase the risk of rejection in low risk matched related donor transplant for thalassemia and this risk can be partially overcome by additional immunosuppression during conditioning. Biology of Blood and Marrow Transplantation,26(10):1886-1893, 2020. https://doi.org/10.1016/j.bbmt.2020.06.013
40. Faulkner L., Verna M., Rovelli A., Kumar Agarwal R., Dhanya R., Ramprakash S., Raghuram CP, Mehta P., Sandeep Elizabeth S., Khalid S., Batool A., Khan-Ghilani S.. Fatima I., Zara T., Marwah P., Soni R., Trivedi D., Conter V., Canesi M., Othman D., Faeq V., Kleinschmidt K., Yesillipek A., Catherine G. Lam C.G., Scott C. Howard S.C. and Corbacioglu S. Setting up and sustaining blood and marrow transplant services for children in Middle-Income Economies: An experience-driven position paper on behalf of the PDWP of the EBMT Bone Marrow Transplantation, (2021) 56:536–543. https://doi.org/10.1038/s41409- 020-0983-5.
41. Faulkner L.: Telemedicine in COVID-19 Era: Lessons from Online Co-Management of Bone Marrow Transplant Patients. Journal of Islamabad Medical & Dental College 9, n. 2 (2020): 79–81. https://doi.org/10.35787/jimdc.v9i2.550.
42. Dhanya R., Agarwal R.K, Sedai A., Kumari A., Parmar L., Hegde S., Gowda A., Gujjal P., Pushpa H, Dasaratha Ramaiah Jinka D.R., Karri, C., Jali S., Tallur N., Shenoy U., Pinto D., Ramprakash S., Raghuram S., Trivedi D., Cao X., and Faulkner L. ‘Life expectancy and risk factors for early death in patients with severe fhalassemia syndromes in South India’. Blood Advances (2020) 4 (7): 1448–1457. doi: 10.1182/bloodadvances.2019000760