Cure2Children Foundation

Firenze, Italy

Cure2Children Foundation

Firenze, Italy
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Agarwal R.K.,Sankalp India Foundation | Kumari A.,Sankalp India Foundation | Sedai A.,Jagriti Innovations | Parmar L.,Sankalp India Foundation | And 2 more authors.
Biology of Blood and Marrow Transplantation | Year: 2017

Three-loci low-resolution (LR) or intermediate-resolution HLA typing is generally considered adequate in the related blood and marrow transplantation (BMT) context. However, a single high-resolution (HR) mismatch may have a similar adverse impact on BMT outcome as an LR one. We sought to determine the frequency of mismatches that may go undetected when standard typing (LR or 3-loci HR) is used compared with 6-loci HR typing for related donor compatibility testing, and to assess its impact on relevant BMT outcomes. We analyzed data from a total of 2554 6-loci (HLA-A, -B, -C, -DRB1, -DQB1, and -DPB1) HR sequence-based typing (full typing [FT]) from 754 patients, 1011 siblings, and 789 parents done at DKMS Germany (www.DKMS.de) between January 1, 2014, and June 21, 2016. We also studied 38 cases in which the family had undergone 3-loci HLA typing (standard typing [ST]). Patients were from India (70%), Pakistan (22%), and Sri Lanka (8%). The IMGT/HLA database (www.ebi.ac.uk/ipd/imgt/hla) was used to tease out nonpermissive DPB1 mismatches. HLA disparity-related outcomes, such as rejection and graft-versus-host disease (GVHD) were assessed in a retrospective matched-pair cohort of 50 patients (25 with ST and 25 with FT) who underwent BMT for severe thalassemia from compatible related donors. We found fully matched (either 12/12 HR matches or with a single permissive DPB1 mismatch) related donors for 285 patients (38%). Of these donors, 89% were siblings and 11% were parents. The likelihood of matching on an individual locus on LR but not on HR was found to be 5%. A total of 9 donors (3%; 7 siblings and 2 parents) who would have been considered a full match by HR typing on A, B, and DRB1 alone were not a match by extended 6-loci HR typing. Five of these 9 donors had a mismatch on C or DQB1, and 4 had a nonpermissive DPB1 mismatch. In this group, 5 donors (56%) belonged to a consanguineous family, in 2 donors (22%) there was no reported consanguinity, and in 2 donors (22%) consanguinity was unknown. We identified 18 donors (6%; 13 siblings and 5 parents) who would have been considered a 12/12 match by LR HLA typing alone but were found not to match on extended HR typing. In this group, 11 donors (61%) were from consanguineous families, 3 donors (17%) had no reported consanguinity, and in 4 donors (22%) consanguinity was unknown. Outcome analysis showed that the actuarial proportion of patients with GVHD was 4% in the FT group compared with 16% in the ST group, with log-rank P = .1952. The ST group included 2 patients with grade III-IV acute GVHD and 1 patient each with moderate and severe chronic GVHD, whereas the FT group only 1 patient with grade III acute GVHD. We conclude that even in the context of related donors, the use of LR and/or 3-loci (A, B, and DRB1) HR HLA typing might result in a sizable risk of missing a clinically relevant mismatch, which may have an adverse impact on transplantation outcomes. In the Indian subcontinent, this observation is not limited to putatively compatible parents or consanguineous families; we recommend full 6-loci HR HLA typing even for matched related BMTs. © 2017 The American Society for Blood and Marrow Transplantation.


Parikh N.S.,Connecticut Children’s Medical Center | Howard S.C.,University of Memphis | Chantada G.,Hospital JP Garrahan | Israels T.,VU University Amsterdam | And 6 more authors.
Pediatric Blood and Cancer | Year: 2015

Neuroblastoma is the most common extracranial solid tumor in childhood in high-income countries (HIC), where consistent treatment approaches based on clinical and tumor biological risk stratification have steadily improved outcomes. However, in low- and middle- income countries (LMIC), suboptimal diagnosis, risk stratification, and treatment may occur due to limited resources and unavailable infrastructure. The clinical practice guidelines outlined in this manuscript are based on current published evidence and expert opinions. Standard risk stratification and treatment explicitly adapted to graduated resource settings can improve outcomes for children with neuroblastoma by reducing preventable toxic death and relapse. © 2015 The Authors.


PubMed | Fred Hutchinson Cancer Research Center, University of Memphis, Childrens Hospital of Rabat, Outreach and 6 more.
Type: Guideline | Journal: Pediatric blood & cancer | Year: 2015

Neuroblastoma is the most common extracranial solid tumor in childhood in high-income countries (HIC), where consistent treatment approaches based on clinical and tumor biological risk stratification have steadily improved outcomes. However, in low- and middle- income countries (LMIC), suboptimal diagnosis, risk stratification, and treatment may occur due to limited resources and unavailable infrastructure. The clinical practice guidelines outlined in this manuscript are based on current published evidence and expert opinions. Standard risk stratification and treatment explicitly adapted to graduated resource settings can improve outcomes for children with neuroblastoma by reducing preventable toxic death and relapse.


Agarwal R.K.,Jagriti Innovations | Sedai A.,Jagriti Innovations | Dhimal S.,Jagriti Innovations | Ankita K.,Jagriti Innovations | And 13 more authors.
Journal of the American Medical Informatics Association | Year: 2014

Jagriti Innovations developed a collaboration tool in partnership with the Cure2Children Foundation that has been used by health professionals in Italy, Pakistan, and India for the collaborative management of patients undergoing bone marrow transplantation (BMT) for thalassemia major since August 2008. This online openaccess database covers data recording, analyzing, and reporting besides enabling knowledge exchange, telemedicine, capacity building, and quality assurance. As of February 2014, over 2400 patients have been registered and 112 BMTs have been performed with outcomes comparable to international standards, but at a fraction of the cost. This approach avoids medical emigration and contributes to local healthcare strengthening and competitiveness. This paper presents the experience and clinical outcomes associated with the use of this platform built using open-source tools and focusing on a locally pertinent tertiary care procedure-BMT.


El Missiry M.,Cure2Children Foundation | Hussein M.H.,Cure2Children Foundation | Khalid S.,Cure2Children Foundation | Yaqub N.,Childrens Hospital Pakistan Institute of Medical science | And 4 more authors.
Anemia | Year: 2014

Zinc (Zn) is essential for appropriate growth and proper immune function, both of which may be impaired in thalassemia children. Factors that can affect serum Zn levels in these patients may be related to their disease or treatment or nutritional causes. We assessed the serum Zn levels of children with thalassemia paired with a sibling. Zn levels were obtained from 30 children in Islamabad, Pakistan. Serum Zn levels and anthropometric data measures were compared among siblings. Thalassemia patients' median age was 4.5 years (range 1-10.6 years) and siblings was 7.8 years (range 1.1-17 years). The median serum Zn levels for both groups were within normal range: 100 μg/dL (10 μg/dL-297 μg/dL) for patients and 92 μg/dL (13 μg/dL-212 μg/dL) for siblings. There was no significant difference between the two groups. Patients' serum Zn values correlated positively with their corresponding siblings (r = 0.635, P < 0.001). There were no correlations between patients' Zn levels, height for age Z-scores, serum ferritin levels, chelation, or blood counts (including both total leukocyte and absolute lymphocyte counts). Patients' serum Zn values correlated with their siblings' values. In this study, patients with thalassemia do not seem to have disease-related Zn deficiency. Copyright © 2014 Mohamed El Missiry et al.


PubMed | Cure2Children Foundation and Childrens Hospital Pakistan Institute of Medical science
Type: | Journal: Anemia | Year: 2014

Zinc (Zn) is essential for appropriate growth and proper immune function, both of which may be impaired in thalassemia children. Factors that can affect serum Zn levels in these patients may be related to their disease or treatment or nutritional causes. We assessed the serum Zn levels of children with thalassemia paired with a sibling. Zn levels were obtained from 30 children in Islamabad, Pakistan. Serum Zn levels and anthropometric data measures were compared among siblings. Thalassemia patients median age was 4.5 years (range 1-10.6 years) and siblings was 7.8 years (range 1.1-17 years). The median serum Zn levels for both groups were within normal range: 100g/dL (10g/dL-297g/dL) for patients and 92g/dL (13g/dL-212g/dL) for siblings. There was no significant difference between the two groups. Patients serum Zn values correlated positively with their corresponding siblings (r = 0.635, P < 0.001). There were no correlations between patients Zn levels, height for age Z-scores, serum ferritin levels, chelation, or blood counts (including both total leukocyte and absolute lymphocyte counts). Patients serum Zn values correlated with their siblings values. In this study, patients with thalassemia do not seem to have disease-related Zn deficiency.


Faulkner L.B.,Cure2Children Foundation | Uderzo C.,Cure2Children Foundation | Uderzo C.,University of Milan Bicocca | Masera G.,University of Milan Bicocca
Journal of Pediatric Hematology/Oncology | Year: 2013

Thalassemia major (TM) is the most frequent life-threatening noninfectious disease of childhood in the Middle East, South Asia, and Pacific Islands where it accounts for a significant proportion of childhood mortality, morbidity, and related health care expenses. In spite of major advances in supportive care during the last decade, many patients in low-income and middle-income countries still fare poorly because of high treatment costs and lack of accessible multidisciplinary teams, not to consider the risk of blood-borne infections, primarily hepatitis C. In selected low-risk patients with a compatible sibling, TM is highly curable by bone marrow transplantation (BMT), which also improves the quality of life and is cost-effective. Starting in 2008, the Cure2Children Foundation (C2C), an Italian Non-Governmental Organization, has supported a BMT network in Pakistan, which during 2012 was extended to India. The primary aim of this project was to assess feasibility, outcomes, and costs of matched-related BMT for thalassemia in young low-risk children using a well-established and tolerable strategy. A total of 100 matched-related BMTs have been performed to date by partner institutions within this C2C-supported network; in the 50 low-risk cases with TM, over 90% disease-free survival was obtained with procedure expenses within 10,000 USD/BMT, that is, an outcome comparable to that obtained in affluent countries but with a fraction of the expenses. This cure rate was also obtained in start-up BMT centers (1 in Pakistan and 1 in India) within a structured and intensive cooperation program. Twinning and other international cooperation strategies based on shared principles and a common vision may substantially facilitate access to BMT. © 2013 Lippincott Williams & Wilkins.


Mehta P.A.,Cincinnati Childrens Hospital Medical Center | Faulkner L.B.,Cure2Children Foundation
Biology of Blood and Marrow Transplantation | Year: 2013

Hematopoietic cell transplantation (HCT) remains the sole available curative option for patients with β-thalassemia major. Expanded and improved supportive therapies for thalassemia now routinely extend the life span of affected individuals well into adulthood. Consequently, in regions of the world where this care is readily available, HCT has been pursued infrequently, in part owing to concerns about an expected lack of balance between risks and benefits. More recently, however, recognition of significant health problems in older patients with thalassemia, along with recognition of increased risks of graft-versus-host disease (GVHD), graft rejection, and impaired organ function leading to inferior HCT outcomes in this particular group, seem to be turning the wheels and tipping the balance again in the direction of consideration for earlier HCTs. In contrast, in countries where thalassemia is most prevalent (>100,000 new children born each year in Middle East and southeast Asia), lack of supportive care standards together with often insufficient access to dedicated health care facilities, results in the majority of these children not reaching adulthood, further supporting the need for expanded access to HCT for these patients. The cost of HCT is equivalent to that of a few years of noncurative supportive care, such that HCT in low-risk young children with a compatible sibling is justified not only medically and ethically but also financially. International cooperation can play a major role in increasing access to safe and affordable HCT in countries where there is a considerable shortage of transplantation centers. In this article, we review the current status of bone marrow transplantation for thalassemia major, with particular emphasis on a global prospective. © 2013 American Society for Blood and Marrow Transplantation.

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