A Single-Center Pilot Prospective Study of Topical Application of Platelet-Derived Eye Drops for Patients with Ocular Chronic Graft-versus-Host Disease.
Ocular involvement of chronic graft-versus-host disease (cGVHD) is a complication that occurs in up to 60% of patients after allogeneic hematopoietic stem cell transplantation. Conventional therapeutic options include medical and surgical procedures that are administered depending on the severity of the condition, but most of them have provided unsatisfactory results and, to date, there is no consensus about treatment. We considered that topical application of a platelet lysate, administered as eye drops, might be considered an alternative worthwhile of investigation to treat ocular surface disorders in patients suffering from cGVHD. Therefore, we conducted a single-center prospective pilot study to assess the efficacy and safety of using eye drops made from reconstituted lysed platelet concentrate. Twenty-six patients with ocular cGVHD were eligible for the study; all but 2 completed their scheduled 1-year treatment and complied with the hematologic and ophthalmic regimen. At their first assessment interviews, after 30 days of treatment, 91% of patients reported an improvement in their symptoms and for 32%, substantive objective differences were measured. Remission of corneal damage was seen for 86% of our cohort, and improved National Institutes of Health scores for 73%, of whom 8% achieved the best score of 0 (ie, non-dry eye). Similar results were seen at later time points. Comparing outcomes for our patient cohort to those determined retrospectively for patients in our institutional database revealed a 5-year overall survival (OS) of 65%. This OS is comparable to patients with limited cGVHD (75%) and is superior to that of patients with nonocular extensive cGVHD or without cGVHD (30% and 59%, respectively) (P = .013). Our results suggest that platelet-derived eye drops are a safe, practical, and well-tolerated therapeutic option that offers substantial benefits for most patients affected by ocular cGVHD at onset. The favorable OS of our patient cohort suggests that this topical therapy, rather than systemic immunosuppression, may be the treatment of choice.
Hematology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy. Electronic address: fazallio@ospedale.al.it.
Francesco
F
Zallio
Transfusion and Regeneration Medicine, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Laura
L
Mazzucco
Hematology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Federico
F
Monaco
Ophthalmology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Maria Rosa
MR
Astori
Division of Nuclear Medicine, San Giovanni Battista Hospital and University of Torino, Torino, Italy.
Roberto
R
Passera
Hematology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Giovanna
G
Drago
Hematology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Stefania
S
Tamiazzo
Pharmacologic Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Manuela
M
Rapetti
Ophthalmology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Daniela
D
Dolcino
Transfusion and Regeneration Medicine, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Roberto
R
Guaschino
Hematology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Massimo
M
Pini
Hematology Department, SS Antonio & Biagio and C. Arrigo Hospital, Alessandria, Italy.
Marco
M
Ladetto
D000287:Administration, Topical; D000328:Adult; D000368:Aged; D001792:Blood Platelets; D002908:Chronic Disease; D015352:Dry Eye Syndromes / Q000188:drug therapy* / Q000209:etiology; D005128:Eye Diseases / Q000188:drug therapy / Q000209:etiology; D005260:Female; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D009883:Ophthalmic Solutions / Q000627:therapeutic use*; D010865:Pilot Projects; D011446:Prospective Studies; D014184:Transplantation, Homologous / Q000009:adverse effects; D055815:Young Adult
D009883:Ophthalmic Solutions
Allogeneic transplantation; Eye drops; Graft-versus-host disease; Lysed platelet concentrate