Radiotherapy, laminectomy, and chemotherapy showed comparable ability to relieve or improve SCC. However, patients treated with chemotherapy usually did not require additional therapy, whereas patients treated either with radiotherapy or laminectomy commonly did. No patient presenting with (or developing) severe motor deficit recovered or improved. Sequelae were documented in 44% of surviving patients.

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 open-access 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.

4331 Since May 2008, the Cure2Children Foundation has supported both financially and professionally a network of centers in Pakistan for stem cell transplantation applied to the cure of thalassemia major, a very prevalent disease with more than 60.000 registered cases in that country. The methodology employed consists of matched-related standard bone marrow transplantation after myeloablative chemotherapy as currently employed at the Mediaterranean Institute of Hematology for younger patients with transfusion-dependent thalassermia (thiotepa 10 mg/kg, busulfan 14 mg/kg and cyclophosphamide 200 mg/kg) followed by GVHD/rejection prophylaxis with prednisone/methotrexate/cyclosporin. Management standards for central venous access, severe pancytopenia, immunosuppression, hospital infection control, and other relevant issues have been addressed by local training as well as with web-based data management and videoconferencing. A total of 15 transplants have been performed to date, 7 at an established center (National Institute of Blood Diseases, Karachi), and 8 at newly developed services (5 at the Pakistan Institute of Medical Sciences, Islamabad, and 3 at Shifa International Hospital, Islamabad). Patient characteristics Median age 3.4 years (range 0.9 to 6.2), 7 males and 8 females. Results At a median follow up of 135 days (range 17-336), actuarial thalassemia-free survival is 79% and overall survival 93%. So far two patients had a graft failure and are alive and well after autologous reconstitution. One patient died for an intracranial bleed, one developed grade 3 acute GVHD (which responded promptly to steroid therapy), 4 had subclinical CMV activation and one developed pulmonary tuberculosis (currently on treatment and doing well). No case of chronic GVHD has been observed so far. Conclusions These preliminary results are comparable to those obtained in western centers, detailed updated clinical and cost analysis will be presented. We believe that this ongoing experience might contribute to extend access to allogeneic transplantation for the cure of low-risk thalassemia children in limited resource settings. Disclosures: No relevant conflicts of interest to declare.

Retinitis Pigmentosa (RP) and Idiopathic Intracranial Hypertension (IIH) are two different pathologies that can have a very similar presentation (headache and loss of visión). The differential diagnosis of both is essential due to their different management and prognosis. We present the case of a young woman with a 2-year history of headache, visual loss and buzzing in the head. In the fundus of both eyes, typical findings of RP (bone spicules in the middle retina and arteriolar thinning) and IIH (bilateral papilledema) stand out, although the latter is also described in cases of RP. After a complete study in Ophthalmology and Neurology, the diagnosis of both pathologies is reached, with symptomatic and visual improvement after starting treatment to reduce intracranial pressure. The coincidence of both entities is extremely infrequent in the same patient, a high index of suspicion is required due to their clinical similarities and a multidisciplinary collaboration for their diagnosis.