A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcers

Lillian McMahon, Hannah Tamary, Melissa Askin, Patricia Adams-Graves, Robert T. Eberhardt, Millicent Sutton, Elizabeth C. Wright, Serguei A. Castaneda, Douglas V. Faller, Susan P. Perrine

Research output: Contribution to journalArticle

43 Citations (Scopus)

Abstract

Sickle cell leg ulcers are often debilitating, refractory to healing, and prone to recurrence. Healing of leg ulcers was incidentally observed during dose-ranging trials of Arginine Butyrate in beta haemoglobinopathies. Here, a controlled Phase II trial was performed in sickle cell patients who had lower extremity ulcers refractory to standard care for at least 6 months. Patients were randomized to receive standard local care alone (Control Arm) or standard care with Arginine Butyrate administered 5 d/week (Treatment Arm), for 12 weeks. Ulcers were photographed weekly, traced, and ulcer areas were calculated by computerized planimetry and compared between the two study arms. Twenty-seven study courses were evaluated. Control Arm subjects had 25 ulcers with a mean area of 25·7 cm2 initially and 23·2 cm2 after 12 weeks; 2/25 (8%) healed completely. Treatment Arm subjects had 37 ulcers with a mean area of 50·6 cm2 initially and 28·3 cm2 at 12 weeks; 11/37 of these (30%) healed completely. After 3 months, proportions of ulcers which healed were 6/25 (24%) and 29/37 (78%), in the Control and Treatment Arms respectively (P < 0·001). These findings strongly suggest that Arginine Butyrate merits further evaluation for the treatment of refractory sickle cell leg ulcers in larger trials.

Original languageEnglish (US)
Pages (from-to)516-524
Number of pages9
JournalBritish Journal of Haematology
Volume151
Issue number5
DOIs
StatePublished - Dec 1 2010

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Leg Ulcer
Ulcer
Arm
Therapeutics
Hemoglobinopathies
arginine butyrate
Lower Extremity
Recurrence

All Science Journal Classification (ASJC) codes

  • Hematology

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A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcers. / McMahon, Lillian; Tamary, Hannah; Askin, Melissa; Adams-Graves, Patricia; Eberhardt, Robert T.; Sutton, Millicent; Wright, Elizabeth C.; Castaneda, Serguei A.; Faller, Douglas V.; Perrine, Susan P.

In: British Journal of Haematology, Vol. 151, No. 5, 01.12.2010, p. 516-524.

Research output: Contribution to journalArticle

McMahon, L, Tamary, H, Askin, M, Adams-Graves, P, Eberhardt, RT, Sutton, M, Wright, EC, Castaneda, SA, Faller, DV & Perrine, SP 2010, 'A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcers', British Journal of Haematology, vol. 151, no. 5, pp. 516-524. https://doi.org/10.1111/j.1365-2141.2010.08395.x
McMahon, Lillian ; Tamary, Hannah ; Askin, Melissa ; Adams-Graves, Patricia ; Eberhardt, Robert T. ; Sutton, Millicent ; Wright, Elizabeth C. ; Castaneda, Serguei A. ; Faller, Douglas V. ; Perrine, Susan P. / A randomized phase II trial of Arginine Butyrate with standard local therapy in refractory sickle cell leg ulcers. In: British Journal of Haematology. 2010 ; Vol. 151, No. 5. pp. 516-524.
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